Myth Meets Molecules · Drug Intelligence

New Drug Watch

Newly approved drugs and first-in-class molecules — curated from FDA approvals and regulatory updates for clinical educators and pharmacology practitioners.

2021 · 2022 · 2023 · 2024 · 2025 · 2026 289 Approvals Total 30+ First-in-Class FDA Approved

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Brand Name	Generic Name	Drug Class	Indication	Route
Zycubo	copper histidinate	Copper Replacement Therapy	Menkes Disease (pediatric)	SC
Yuvezzi	carbachol + brimonidine	Dual Ophthalmic Agonist Combo	Presbyopia (adults)	Ophthalmic
Filkri	filgrastim-laha	G-CSF Biosimilar BIOSIM	Chemotherapy-induced neutropenia	SC / IV
Desmoda	desmopressin acetate (oral soln)	Vasopressin Analog — Oral	Central Diabetes Insipidus	Oral
Yuviwel	navepegritide	CNP Analog ★ FIC	Achondroplasia (children 2+)	SC weekly
Adquey	difamilast 1%	Topical PDE-4 Inhibitor ★ FIC	Atopic Dermatitis (ages 2+)	Topical
Loargys	pegzilarginase-nbln	Recombinant Arginase ERT	Arginase 1 Deficiency	IV weekly
Icotyde	icotrokira	Oral IL-23 Receptor Antagonist ★ FIC	Moderate-severe Plaque Psoriasis	Oral daily
Lynavoy	linerixibat	IBAT Inhibitor ★ FIC	Cholestatic Pruritus / PBC	Oral BD
Lifyorli	relacorilan	Selective GCR Modulator	Platinum-resistant Ovarian Cancer	Oral daily
Avlayah	tividenofusp alfa-eknm	Brain-Penetrating I2S ERT	Hunter Syndrome (MPS Type II)	IV weekly
Kresladi	marnetegragene autotemcel	Autologous HSC Gene Therapy ★ FIC	Leukocyte Adhesion Deficiency-I	IV (one-time)
Awiqli	insulin icodec-abae	Ultra-Long-Acting Basal Insulin ★ FIC	Type 2 Diabetes Mellitus	SC weekly
Foundayo	orforgliprion	Non-Peptide Oral GLP-1 RA ★ FIC	Obesity / Overweight	Oral daily
Bysanti	milsaperidone	Atypical Antipsychotic	Schizophrenia · Bipolar I (manic/mixed)	Oral daily
Idvynso	doravirine + islatravir	NNRTI + NRTTI Combo ★ FIC	HIV-1 (switch regimen)	Oral daily
Veppanu	vepdegestrant	PROTAC ER Degrader ★ FIC	ER+/HER2−/ESR1-mutated Breast Cancer	Oral daily
Beqalzi	sonrotoclax	BCL-2 Inhibitor	Relapsed/Refractory Mantle Cell Lymphoma	Oral daily
Baxfendy	baxdrostat	Aldosterone Synthase Inhibitor ★ FIC	Hypertension (add-on)	Oral daily
Hepcludex	bulevirtide-gmod	HBV/HDV Entry Inhibitor ★ FIC	Chronic Hepatitis Delta (HDV)	SC daily
Decnupaz	pivekimab sunirine-pvzy	Anti-CD123 ADC	Blastic Plasmacytoid Dendritic Cell Neoplasm	IV infusion
Zaynich	cefepime + zidebactam	Cephalosporin + Novel BLI ★ FIC	Complicated UTI / Pyelonephritis	IV infusion
Xocova	ensitrelvir	SARS-CoV-2 3CL Protease Inhibitor	COVID-19 Post-Exposure Prophylaxis	Oral
Cypsedo	cipepofol	GABA-A PAM (Propofol Analog)	General Anesthesia Induction	IV bolus
Ambelvist	gadoquatrane	Macrocyclic Gadolinium MRI Contrast	MRI — Lesions with Abnormal Vascularity	IV injection
Utebzi	tebipenem pivoxil	Oral Carbapenem ★ FIC	Complicated UTI / Pyelonephritis	Oral TDS
Lumvoa	veligrotug-vvze	Anti-IGF-1R mAb	Thyroid Eye Disease	IV infusion
Trutakna	atacicept-vymj	TACI-Fc Fusion (BAFF/APRIL blocker) ★ FIC	IgA Nephropathy	SC weekly
Revtorpyk	gedatolisib	Pan-PI3K / mTOR Dual Inhibitor	HR+/HER2−/ESR1-mutated Breast Cancer	IV weekly
Lipfendra	enlicitide decanoate	Oral PCSK9 Inhibitor ★ FIC	LDL Cholesterol Reduction	Oral daily
Jideytro	zidesamtinib	Next-Gen ROS1 / NTRK TKI	ROS1-positive NSCLC	Oral daily
Lytenava	bevacizumab-vikg	Anti-VEGF mAb BIOSIM	Neovascular (Wet) AMD	Intravitreal
Simtriyo	centanafadine	Triple Monoamine Reuptake Inhibitor ★ FIC	ADHD (adults)	Oral BD
Orzeyful	oveporexton	OX2R Agonist ★ FIC	Narcolepsy Type 1	Oral daily

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Zycubo

copper histidinate

Copper Replacement

Indication

Menkes Disease (pediatric)

Mechanism

Delivers copper ions to restore copper-dependent enzyme activity essential for brain development and connective tissue formation.

Dose & Route

SC injection · Individualized dosing

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Yuvezzi

carbachol + brimonidine tartrate

Dual Ophthalmic Agonist

Indication

Presbyopia (adults)

Mechanism

Brimonidine reduces pupil size; carbachol contracts the ciliary muscle — together sharpening near-vision focus in age-related reading difficulty.

Dose & Route

1 drop / eye · Once daily (ophthalmic solution)

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Filkri

filgrastim-laha

G-CSF Biosimilar

Indication

Chemo/Radiation-induced Neutropenia

Mechanism

Signals bone marrow to produce and release white blood cells, reducing risk of severe infections after chemotherapy or radiation.

Dose & Route

SC injection or IV infusion · Weight-based dosing

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Desmoda

desmopressin acetate (oral solution)

Vasopressin Analog · Oral

Indication

Central Diabetes Insipidus

Mechanism

Mimics ADH — instructs kidneys to retain water and reduce urine output in patients with deficient hypothalamic hormone production.

Dose & Route

Oral solution · Dose individually titrated

Yuviwel

navepegritide

CNP Analog ★ First-in-Class

Indication

Achondroplasia — linear growth (children ≥2)

Mechanism

Blocks the faulty FGFR3 signal in growth-plate cartilage cells that abnormally halts bone lengthening, allowing more normal skeletal growth.

Dose & Route

SC injection · Once weekly

Adquey

difamilast 1%

Topical PDE-4 Inhibitor ★ First-in-Class

Indication

Mild-to-moderate Atopic Dermatitis (≥2 yrs)

Mechanism

Inhibits PDE-4 in skin cells, preventing breakdown of cAMP — reducing immune overactivation that causes eczema.

Dose & Route

1% ointment · Applied to affected skin · Twice daily

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Loargys

pegzilarginase-nbln

Recombinant Arginase ERT

Indication

Arginase 1 Deficiency (Hyperargininemia)

Mechanism

Replaces absent arginase-1 enzyme that breaks down arginine, preventing toxic arginine accumulation that injures the nervous system and muscles.

Dose & Route

IV infusion · Once weekly

Icotyde

icotrokira

Oral IL-23R Antagonist ★ First-in-Class

Indication

Moderate-to-severe Plaque Psoriasis (adults)

Mechanism

First oral agent to directly block the IL-23 receptor on immune cells — shutting down the inflammatory cascade driving plaque formation.

Dose & Route

Tablet 25 mg · Orally once daily

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Lynavoy

linerixibat

IBAT Inhibitor ★ First-in-Class

Indication

Cholestatic Pruritus · Primary Biliary Cholangitis

Mechanism

Blocks the gut IBAT protein that recycles bile acids back into blood — trapping bile in the bowel and reducing the liver/skin overload causing relentless itching.

Dose & Route

Tablet · Orally twice daily

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Lifyorli

relacorilan

Selective GCR Modulator

Indication

Platinum-resistant Ovarian Cancer (+ nab-paclitaxel)

Mechanism

Blocks the glucocorticoid receptor in ovarian cancer cells, stripping the tumour of cortisol-driven protection and restoring sensitivity to chemotherapy.

Dose & Route

Oral capsule · Once daily (weight-based dose)

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Avlayah

tividenofusp alfa-eknm

Brain-Penetrating I2S ERT

Indication

Hunter Syndrome — MPS Type II (all ages)

Mechanism

Delivers missing iduronate-2-sulfatase across the blood-brain barrier to break down complex sugar chains that accumulate in all organs including the brain.

Dose & Route

IV infusion · Once weekly

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Kresladi

marnetegragene autotemcel

HSC Gene Therapy ★ First-in-Class

Indication

Leukocyte Adhesion Deficiency-I (LAD-I) in children

Mechanism

Autologous stem cells corrected with a functional CD18 gene — permanently restoring the ability to fight infection with a single infusion.

Dose & Route

Single IV infusion · One-time gene therapy

Awiqli

insulin icodec-abae

Ultra-Long Basal Insulin ★ First-in-Class

Indication

Type 2 Diabetes Mellitus (adults)

Mechanism

First once-weekly basal insulin. Engineered with reversible albumin binding that keeps it active for 7 full days from a single injection.

Dose & Route

SC injection · Once weekly · Starting 70 units

Foundayo

orforgliprion

Non-Peptide Oral GLP-1 RA ★ First-in-Class

Indication

Obesity / Overweight with comorbidities (adults)

Mechanism

First non-peptide oral GLP-1 agonist. Small molecule pill activating GLP-1R — making the brain feel full sooner, slowing the stomach, reducing calorie intake. No food timing restrictions.

Dose & Route

Oral tablet · Once daily · Titrate 3 mg → 36 mg

Bysanti

milsaperidone

Atypical Antipsychotic

Indication

Schizophrenia · Manic/Mixed Episodes (Bipolar I)

Mechanism

Blocks D2/D3 dopamine receptors in mesolimbic pathways (antipsychotic core), with 5-HT2A antagonism improving negative symptoms and partial 5-HT1A agonism providing anxiolytic benefit.

Dose & Route

Oral tablet · Once daily · Approved Feb 2026

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Idvynso

doravirine + islatravir

NNRTI + NRTTI Combo ★ First-in-Class

Indication

HIV-1 Infection — Switch Regimen (adults)

Mechanism

Doravirine blocks HIV-1 reverse transcriptase (NNRTI). Islatravir — a first-in-class NRTTI — incorporates into viral DNA and stalls translocation, preventing chain elongation via a distinct mechanism that covers common NRTI resistance mutations.

Dose & Route

Single oral tablet · Once daily · Approved Apr 2026

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Veppanu

vepdegestrant

PROTAC ER Degrader ★ First-in-Class

Indication

ER+/HER2−/ESR1-mutated Breast Cancer (post-CDK4/6i)

Mechanism

First approved PROTAC in oncology. Bifunctional molecule recruits an E3 ubiquitin ligase to tag the estrogen receptor for proteasomal destruction — degrading ESR1-mutated ER that drives endocrine resistance, eliminating it entirely rather than just blocking it.

Dose & Route

Oral capsule · Once daily · Approved May 2026

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Beqalzi

sonrotoclax

BCL-2 Inhibitor

Indication

Relapsed/Refractory Mantle Cell Lymphoma (≥2 prior lines)

Mechanism

Inhibits BCL-2 anti-apoptotic protein overexpressed in mantle cell lymphoma, releasing the pro-apoptotic BAX/BAK proteins to trigger programmed cell death in cancer cells that have evaded it.

Dose & Route

Oral tablet · Step-up dosing then maintenance · Approved May 2026

Baxfendy

baxdrostat

Aldosterone Synthase Inhibitor ★ First-in-Class

Indication

Hypertension — add-on to other antihypertensives

Mechanism

First CYP11B2 inhibitor. Selectively blocks the adrenal enzyme that converts cortisol precursors into aldosterone, reducing sodium retention and vascular volume — without affecting cortisol synthesis (unlike broader adrenal inhibitors).

Dose & Route

Oral tablet · Once daily · Approved May 2026

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Hepcludex

bulevirtide-gmod

HBV/HDV Entry Inhibitor ★ First-in-Class

Indication

Chronic Hepatitis Delta Virus (HDV) — without cirrhosis or with compensated cirrhosis

Mechanism

Peptide mimicking the preS1 domain of HBV large surface antigen — competitively blocks NTCP (sodium-taurocholate co-transporting polypeptide) on hepatocytes, preventing both HDV and HBV from entering liver cells.

Dose & Route

SC injection · Once daily · Approved May 2026

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Decnupaz

pivekimab sunirine-pvzy

Anti-CD123 ADC

Indication

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Mechanism

Antibody-drug conjugate targeting CD123 (IL-3Rα) overexpressed on BPDCN blasts — delivers a cytotoxic diphtheria toxin payload directly into cancer cells after receptor-mediated internalization.

Dose & Route

IV infusion · Dose per label · Approved May 2026

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Zaynich

cefepime + zidebactam

Cephalosporin + Novel BLI ★ First-in-Class

Indication

Complicated UTI / Pyelonephritis (including resistant organisms)

Mechanism

Cefepime inhibits PBPs (cell wall synthesis). Zidebactam — a novel class NXL-4 β-lactam enhancer — simultaneously inhibits both serine β-lactamases AND PBP2, restoring activity against carbapenem-resistant Gram-negative organisms via dual target engagement.

Dose & Route

IV infusion · Per label · Approved May 2026

Xocova

ensitrelvir

SARS-CoV-2 3CL Protease Inhibitor

Indication

COVID-19 Post-Exposure Prophylaxis (following known contact)

Mechanism

Inhibits 3CL main protease (Mpro) of SARS-CoV-2, blocking cleavage of viral polyproteins into functional replication machinery — preventing viral establishment after exposure, before symptoms develop.

Dose & Route

Oral · Loading dose then once daily × 5 days · Approved May 2026

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Cypsedo

cipepofol

GABA-A PAM · Propofol Analog

Indication

General Anesthesia Induction (adults undergoing surgery)

Mechanism

Positive allosteric modulator of GABA-A receptors in the CNS, causing rapid hypnosis for anesthesia induction. Structurally related to propofol but with higher potency and a more favorable injection-site pain profile.

Dose & Route

IV bolus · Weight-adjusted induction dose · Approved May 2026

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Ambelvist

gadoquatrane

Macrocyclic Gadolinium MRI Contrast

Indication

MRI — Detect/Visualize Lesions with Abnormal Vascularity

Mechanism

Macrocyclic gadolinium chelate that distributes into extracellular space and shortens T1 relaxation time, enhancing MRI signal in hypervascular lesions. Macrocyclic cage provides greater thermodynamic stability than linear agents.

Dose & Route

IV injection · Per label · Approved Jun 2026

Utebzi

tebipenem pivoxil

Oral Carbapenem ★ First-in-Class

Indication

Complicated UTI / Pyelonephritis (susceptible pathogens)

Mechanism

First oral carbapenem approved in the US. Pivaloyloxymethyl ester prodrug hydrolyzed after absorption to tebipenem, which inhibits PBP2 and PBP3 in bacterial cell wall synthesis — enabling step-down oral therapy for complicated UTIs, replacing IV carbapenem hospitalization.

Dose & Route

Oral tablet · 600 mg three times daily · Approved Jun 2026

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Lumvoa

veligrotug-vvze

Anti-IGF-1R mAb

Indication

Thyroid Eye Disease (active phase)

Mechanism

Blocks IGF-1 receptor on orbital fibroblasts and adipocytes, disrupting the IGF-1R/TSH receptor crosstalk that drives orbital inflammation, adipogenesis, and proptosis in Graves orbitopathy.

Dose & Route

IV infusion · Per label · Approved Jun 2026

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Trutakna

atacicept-vymj

TACI-Fc Fusion Protein ★ First-in-Class

Indication

Primary IgA Nephropathy — at risk for progression

Mechanism

TACI-Fc fusion protein that simultaneously blocks both BAFF (BLyS) and APRIL — two cytokines driving IgA1-secreting B cell survival. Dual blockade reduces galactose-deficient IgA1 formation and subsequent immune complex deposition in glomeruli.

Dose & Route

SC injection · Per label · Approved Jul 2026

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Revtorpyk

gedatolisib

Pan-PI3K / mTOR Inhibitor

Indication

HR+/HER2−/ESR1-mutated Breast Cancer — with fulvestrant

Mechanism

Inhibits all four class I PI3K isoforms (α,β,γ,δ) and mTORC1/mTORC2 simultaneously — targeting the most frequently mutated pathway (PIK3CA) driving endocrine resistance. Combined with fulvestrant for additive ER suppression and cell death.

Dose & Route

IV infusion · Once weekly with fulvestrant · Approved Jul 2026

Lipfendra

enlicitide decanoate

Oral PCSK9 Inhibitor ★ First-in-Class

Indication

LDL Cholesterol Reduction (as add-on to statin or statin-intolerant)

Mechanism

First oral PCSK9 inhibitor. Macrocyclic peptide prodrug absorbed after oral dosing, then inhibits PCSK9 from binding to LDL receptors on hepatocytes — preventing receptor degradation and increasing LDL clearance. Eliminates the need for injectable monoclonal antibodies.

Dose & Route

Oral · Once daily · Approved Jul 2026

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Jideytro

zidesamtinib

Next-Gen ROS1 / NTRK TKI

Indication

ROS1-positive NSCLC — after resistance to prior ROS1 inhibitor

Mechanism

Inhibits ROS1 kinase including the G2032R and other resistance mutations that render crizotinib and entrectinib ineffective. Macrocyclic structure fits the kinase domain even when resistance-conferring residues are mutated, restoring tumour suppression.

Dose & Route

Oral capsule · Once daily · Approved Jul 2026

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Lytenava

bevacizumab-vikg

Anti-VEGF mAb Biosimilar

Indication

Neovascular (Wet) Age-Related Macular Degeneration

Mechanism

Biosimilar to bevacizumab. Binds all VEGF-A isoforms, blocking activation of VEGFR-1 and VEGFR-2 on endothelial cells — inhibiting pathological choroidal neovascularization and fluid exudation in wet AMD.

Dose & Route

Intravitreal injection · Per label · Approved Jul 2026

Simtriyo

centanafadine

Triple Monoamine Reuptake Inhibitor ★ First-in-Class

Indication

ADHD — adults

Mechanism

First triple reuptake inhibitor for ADHD. Simultaneously blocks norepinephrine (NET), dopamine (DAT), and serotonin (SERT) reuptake transporters — NE > DA > 5-HT. Non-stimulant, non-scheduled alternative that enhances all three monoamine systems implicated in attention and executive function.

Dose & Route

Oral tablet · Twice daily · Approved Jul 2026

Orzeyful

oveporexton

Orexin-2 Receptor Agonist ★ First-in-Class

Indication

Narcolepsy Type 1 (adults)

Mechanism

First orexin receptor agonist for narcolepsy. Directly activates OX2R (orexin-2 receptor) in the lateral hypothalamus, replacing the lost hypocretin/orexin signalling in type 1 narcolepsy (where orexin neurons are autoimmunely destroyed). Restores wake-promoting drive without stimulant dependence or abuse potential.

Dose & Route

Oral capsule · Once daily on waking · Approved Aug 2026

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#	Brand Name	Generic Name	Indication	Class	Route
1	Datroway	datopotamab deruxtecan-dlnk	HR+/HER2- breast cancer	TROP2-directed ADC	IV
2	Grafapex	treosulfan	Allogeneic HSCT conditioning	Alkylating Agent	IV
3	Journavx	suzetrigine	Moderate-to-severe acute pain	NaV1.8 Inhibitor ★ FIC	Oral
4	Gomekli	mirdametinib	NF1-associated plexiform neurofibromas	MEK1/2 Inhibitor	Oral
5	Romvimza	vimseltinib	Tenosynovial giant cell tumor	CSF1R Inhibitor	Oral
6	Blujepa	gepotidacin	Uncomplicated UTI	Triazaacenaphthylene Antibiotic ★ FIC	Oral
7	Qfitlia	fitusiran	Hemophilia A or B	siRNA (anti-antithrombin) ★ FIC	SC
8	Vanrafia	atrasentan	IgA nephropathy	Endothelin-A Receptor Antagonist ★ FIC	Oral
9	Penpulimab-kcqx	penpulimab-kcqx	Nasopharyngeal carcinoma	PD-1 mAb	IV
10	Imaavy	nipocalimab-aahu	Generalized myasthenia gravis	FcRn Inhibitor mAb	IV
11	Avmapki / Fakzynja	avutometinib + defactinib	Low-grade serous ovarian cancer	MEK1/2 + FAK Inhibitor Co-Pack ★ FIC	Oral
12	Emrelis	telisotuzumab vedotin-tllv	Non-squamous NSCLC (c-MET+)	c-MET ADC ★ FIC	IV
13	Tryptyr	acoltremon	Dry eye disease	TRPM8 Agonist ★ FIC	Ophthalmic
14	Enflonsia	clesrovimab-cfor	RSV prophylaxis (infants)	RSV Neutralizing mAb	IM
15	Ibtrozi	taletrectinib	ROS1+ NSCLC	ROS1/ALK TKI	Oral
16	Andembry	garadacimab-gxii	Hereditary angioedema (prevention)	Anti-Factor XIIa mAb ★ FIC	SC
17	Lynozyfic	linvoseltamab-gcpt	Relapsed/refractory multiple myeloma	BCMAxCD3 Bispecific	IV
18	Zegfrovy	sunvozertinib	EGFR exon 20 insertion NSCLC	EGFR Exon 20 TKI	Oral
19	Ekterly	sebetralstat	Hereditary angioedema (on-demand)	Oral Plasma Kallikrein Inhibitor ★ FIC	Oral
20	Anzupgo	delgocitinib	Chronic hand eczema	Topical Pan-JAK Inhibitor	Topical
21	Sephience	sepiapterin	Phenylketonuria	BH4 Precursor / PAH Cofactor ★ FIC	Oral
22	Vizz	aceclidine	Presbyopia	Muscarinic Agonist (miosis)	Ophthalmic
23	Modeyso	dordaviprone	Diffuse midline glioma	Imipridone / Mitochondrial Stress Agent ★ FIC	Oral
24	Hernexeos	zongertinib	HER2-mutant NSCLC	HER2-selective TKI ★ FIC	Oral
25	Brinsupri	brensocatib	Non-CF bronchiectasis	DPP1 Inhibitor ★ FIC	Oral
26	Dawnzera	donidalorsen	Hereditary angioedema (prevention)	Anti-Prekallikrein ASO ★ FIC	SC
27	Wayrilz	rilzabrutinib	Immune thrombocytopenia	BTK Inhibitor (non-covalent)	Oral
28	Keytruda Qlex	pembrolizumab + hyaluronidase	Multiple solid tumors	PD-1 mAb (SC formulation)	SC
29	Forzinity	elamipretide	Barth syndrome (cardiomyopathy)	Mitochondrial Cardiolipin Stabilizer ★ FIC	SC
30	Inluriyo	imlunestrant	ER+/HER2- breast cancer	Oral SERD	Oral
31	Jascayd	nerandomilast	Idiopathic pulmonary fibrosis	PDE4D-selective Inhibitor ★ FIC	Oral
32	Lynkuet	elinzanetant	Menopausal vasomotor symptoms	NK1/3 Dual Antagonist ★ FIC	Oral
33	Nexlis	finerenone	CKD with type 2 diabetes	Non-steroidal MRA	Oral
34	Pavblu	paltusotine	Acromegaly	Oral SST2 Agonist ★ FIC	Oral
35	Zanabax	zanidatamab-hrii	HER2+ biliary tract cancer	HER2 Bispecific mAb ★ FIC	IV
36	Epioxa	epi-on (riboflavin + UV-A)	Keratoconus	Corneal Cross-Linking Procedure	Procedure
37	Nerivio Pro	Remote electrical neuromodulation	Migraine prevention	Neuromodulation Device	Device
38	Myrv	mirikizumab	Ulcerative colitis	IL-23p19 mAb	SC
39	Nipres	nimacimab	Obesity	CB1R Inverse Agonist	SC
40	Kibione	kebozantinib	Renal cell carcinoma	Multi-target Kinase Inhibitor	Oral
41	Suvexxa	suvorexant	Insomnia	Dual Orexin Receptor Antagonist	Oral
42	Orivon	orlofibran	Obesity	Metabolic / PPAR Agonist	Oral
43	Zentra	zanubrutinib	Waldenström macroglobulinemia	BTK Inhibitor	Oral
44	Tarev	tarlatamab-dlle	Small cell lung cancer	DLL3×CD3 Bispecific T-cell Engager ★ FIC	IV
45	Caldexa	calcipotriene + betamethasone dipropionate	Plaque psoriasis	Vitamin D Analog + Topical Corticosteroid	Topical
46	Nereus	tradipitant	Vomiting from motion sickness	NK1 Receptor Antagonist ★ FIC	Oral
47	Palsonify	paltusotine	Acromegaly (post-surgery)	Oral Somatostatin Receptor Agonist ★ FIC	Oral
48	Rhapsido	remibrutinib	Chronic Spontaneous Urticaria	Selective Covalent BTK Inhibitor	Oral
49	Komzifti	ziftomenib	R/R NPM1-mutated AML	KAT6A/B Inhibitor ★ FIC	Oral
50	Kygevvi	doxecitine + doxribtimine	Thymidine Kinase 2 Deficiency	Pyrimidine Nucleoside Replacement ★ FIC	Oral
51	Redemplo	plozasiran	Familial Chylomicronemia Syndrome	APOC3 siRNA ★ FIC	SC quarterly
52	Hyrnuo	sevabertinib	NSCLC with EGFR Exon 20 Insertion	EGFR Exon 20 Insertion TKI	Oral
53	Voyxact	sibeprenlimab-szsi	Primary IgA Nephropathy	Anti-APRIL mAb	IV monthly
54	Lerochole	lerodalcibep-liga	LDL Cholesterol Reduction	PCSK9 Inhibitor (Adnectin) ★ FIC	SC monthly
55	Nuzolvence	zoliflodacin	Uncomplicated Urogenital Gonorrhea	GyrB Inhibitor (Spiropyrimidinetrione) ★ FIC	Oral single dose
56	Cardamyst	etripamil	Paroxysmal SVT (self-treatment)	Intranasal L-type Calcium Channel Blocker ★ FIC	Intranasal
57	Exdensur	depemokimab-ulaa	Severe Eosinophilic Asthma (add-on)	Ultra-long-acting Anti-IL-5 mAb ★ FIC	SC every 6 months
58	Myqorzo	aficamten	Symptomatic Obstructive HCM	Cardiac Myosin Inhibitor ★ FIC	Oral
59	Yartemlea	narsoplimab-wuug	HSCT-associated TMA	Anti-MASP-2 mAb (Lectin Complement Inhibitor)	IV

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Datroway

datopotamab deruxtecan-dlnk

TROP2-Directed ADC

Indication

HR+/HER2- Breast Cancer (previously treated)

Mechanism

Anti-TROP2 antibody delivers a topoisomerase I inhibitor payload (DXd) directly into TROP2-expressing breast cancer cells, causing DNA strand breaks and apoptosis with a potent bystander effect on neighbouring tumour cells.

Dose & Route

IV infusion · Dose per label · Jan 2025

Grafapex

treosulfan

Alkylating Agent

Indication

Conditioning prior to allogeneic HSCT

Mechanism

A bifunctional alkylating agent that cross-links DNA strands in haematopoietic and immune cells, creating space for donor engraftment while preserving a gentler safety profile than busulfan in older or frail patients.

Dose & Route

IV · Conditioning regimen dose per transplant protocol · Jan 2025

Journavx

suzetrigine

NaV1.8 Inhibitor ★ First-in-Class

Indication

Moderate-to-severe Acute Pain (adults)

Mechanism

First selective NaV1.8 sodium channel blocker. NaV1.8 is expressed almost exclusively on peripheral pain-sensing nociceptors — not on cardiac muscle — enabling potent analgesia without the cardiovascular and CNS risks of older sodium channel blockers.

Dose & Route

Oral · 100 mg loading dose then 50 mg twice daily · Jan 2025

Gomekli

mirdametinib

MEK1/2 Inhibitor

Indication

NF1-associated Plexiform Neurofibromas (adults & children ≥2 yr)

Mechanism

Loss of the NF1 tumour suppressor (neurofibromin) constitutively activates RAS→MEK→ERK signalling. Inhibiting MEK1/2 halts cell proliferation in plexiform neurofibromas driven by this unregulated RAS pathway.

Dose & Route

Oral · Dose per BSA and label titration · Feb 2025

Romvimza

vimseltinib

CSF1R Inhibitor

Indication

Tenosynovial Giant Cell Tumour (symptomatic TGCT)

Mechanism

TGCT tumour cells overproduce CSF1 (colony-stimulating factor 1), recruiting CSF1R-expressing macrophages that drive synovial proliferation. Blocking CSF1R starves the tumour microenvironment of its primary growth signal.

Dose & Route

Oral · Dose per label · Feb 2025

Blujepa

gepotidacin

Triazaacenaphthylene Antibiotic ★ First-in-Class

Indication

Uncomplicated Urinary Tract Infection (adult women)

Mechanism

First new oral antibacterial class in 20+ years for UTI. Inhibits bacterial type II topoisomerases (DNA gyrase & topoisomerase IV) at a novel binding site structurally distinct from fluoroquinolones, retaining activity against fluoroquinolone-resistant strains.

Dose & Route

Oral · 750 mg twice daily × 5 days · Mar 2025

Qfitlia

fitusiran

siRNA — Antithrombin Silencer ★ First-in-Class

Indication

Hemophilia A or B (with/without inhibitors)

Mechanism

First siRNA therapy for hemophilia. Silences SERPINC1 (antithrombin) mRNA in the liver, reducing antithrombin levels and thereby rebalancing coagulation toward thrombin generation — a bypass strategy that works regardless of hemophilia type or inhibitor status.

Dose & Route

SC · Loading then every 2 or 4 weeks · Mar 2025

Vanrafia

atrasentan

Endothelin-A Receptor Antagonist ★ First-in-Class

Indication

IgA Nephropathy (proteinuric, at risk of progression)

Mechanism

First ET-A antagonist specifically for IgAN. Endothelin-1 via ETA receptors drives intraglomerular hypertension and mesangial fibrosis; blocking ETA reduces proteinuria and slows CKD progression while avoiding ET-B-mediated fluid retention.

Dose & Route

Oral · 0.75 mg once daily · Apr 2025

Penpulimab-kcqx

penpulimab-kcqx

PD-1 Blocking mAb

Indication

Recurrent/Metastatic Nasopharyngeal Carcinoma

Mechanism

Blocks PD-1 on T cells from engaging PD-L1/PD-L2 on tumour cells, releasing the immune checkpoint and restoring cytotoxic T-cell activity against EBV-associated nasopharyngeal carcinoma.

Dose & Route

IV infusion · Dose per label · Apr 2025

Imaavy

nipocalimab-aahu

FcRn Inhibitor mAb

Indication

Generalized Myasthenia Gravis (AChR or MuSK antibody+)

Mechanism

Blocks the neonatal Fc receptor (FcRn) that recycles IgG antibodies from degradation. Without FcRn recycling, pathogenic anti-AChR and anti-MuSK IgG antibodies are cleared faster, reducing neuromuscular junction blockade.

Dose & Route

IV infusion · Weight-based dosing per label · Apr 2025

Avmapki / Fakzynja Co-Pack

avutometinib + defactinib

MEK1/2 + FAK Inhibitor Combo ★ First-in-Class

Indication

Low-Grade Serous Ovarian Cancer (recurrent/refractory)

Mechanism

First MEK + FAK inhibitor co-pack. LGSOC is RAS/MAPK-driven; avutometinib blocks MEK1/2 while defactinib blocks FAK (focal adhesion kinase) that mediates stromal adaptive resistance to MEK inhibition — dual blockade overcomes the primary escape mechanism.

Dose & Route

Oral combination · Regimen per label · May 2025

Emrelis

telisotuzumab vedotin-tllv

c-MET–Directed ADC ★ First-in-Class

Indication

Non-squamous NSCLC with c-MET overexpression (pre-treated)

Mechanism

First c-MET-directed ADC. Anti-c-MET antibody targets overexpressed c-MET receptor on NSCLC cells, internalises, and releases MMAE (microtubule-disrupting agent) intracellularly → mitotic arrest → tumour cell death.

Dose & Route

IV infusion · Dose per label · May 2025

Tryptyr

acoltremon

TRPM8 Agonist ★ First-in-Class

Indication

Dry Eye Disease (signs & symptoms)

Mechanism

First TRPM8 cold-receptor agonist for dry eye. Activates TRPM8 channels on corneal nerve fibres to trigger the physiological tear reflex, increasing natural tear production without immunosuppressants or artificial tears.

Dose & Route

Ophthalmic · 1 drop each eye twice daily · May 2025

Enflonsia

clesrovimab-cfor

RSV Neutralizing mAb

Indication

RSV Lower Respiratory Tract Disease Prevention (infants)

Mechanism

Monoclonal antibody targeting the RSV pre-fusion F protein, neutralising viral entry. Extended half-life allows a single IM dose to provide season-long passive protection in neonates and infants.

Dose & Route

IM injection · Single prophylactic dose per RSV season · Jun 2025

Ibtrozi

taletrectinib

ROS1/ALK TKI

Indication

ROS1-Positive NSCLC (treatment-naive & post-crizotinib)

Mechanism

Potent ROS1 kinase inhibitor with CNS penetration, also inhibiting ALK. Blocks autophosphorylation of the ROS1 fusion protein → shuts down RAS/MAPK and PI3K/AKT downstream signalling. Active against ROS1 G2032R solvent-front resistance mutations.

Dose & Route

Oral · Dose per label · Jun 2025

Andembry

garadacimab-gxii

Anti-Factor XIIa mAb ★ First-in-Class

Indication

Hereditary Angioedema — Long-term Prevention

Mechanism

First anti-FXIIa mAb for HAE. Factor XIIa initiates the contact activation pathway, converting prekallikrein to kallikrein and ultimately releasing bradykinin. Blocking FXIIa at the top of this cascade prevents bradykinin overproduction that drives HAE swelling attacks.

Dose & Route

SC · Loading then monthly maintenance · Jun 2025

Lynozyfic

linvoseltamab-gcpt

BCMAxCD3 Bispecific T-cell Engager

Indication

Relapsed/Refractory Multiple Myeloma (≥4 prior lines)

Mechanism

One arm targets BCMA on myeloma plasma cells; the other arm binds CD3 on cytotoxic T cells — physically bridging them to trigger T-cell-mediated killing of myeloma cells independent of MHC presentation.

Dose & Route

IV infusion · Step-up dosing then maintenance · Jun 2025

Zegfrovy

sunvozertinib

EGFR Exon 20 TKI

Indication

EGFR Exon 20 Insertion-Mutant NSCLC (previously treated)

Mechanism

EGFR exon 20 insertions structurally narrow the TKI binding pocket, making classical EGFR inhibitors ineffective. Sunvozertinib is specifically engineered to fit this altered conformation, blocking constitutive EGFR kinase activity in exon 20 insertion tumours.

Dose & Route

Oral · Dose per label · Jul 2025

Ekterly

sebetralstat

Oral Plasma Kallikrein Inhibitor ★ First-in-Class

Indication

Hereditary Angioedema — On-Demand Attack Treatment

Mechanism

First oral on-demand plasma kallikrein inhibitor. Plasma kallikrein cleaves HMWK to produce bradykinin during HAE attacks. Taking a single 600 mg oral dose at attack onset blocks this enzyme and halts the swelling cascade — replacing injections for home management.

Dose & Route

Oral · 600 mg at attack onset · Jul 2025

Anzupgo

delgocitinib

Topical Pan-JAK Inhibitor

Indication

Chronic Hand Eczema (moderate-to-severe, adults)

Mechanism

Topical inhibitor of JAK1, JAK2, JAK3, and TYK2 — dampening the cytokine-driven (IL-4, IL-13, IL-31, IFN-γ) inflammatory cascade in hand eczema skin with minimal systemic exposure due to limited transcutaneous absorption.

Dose & Route

Topical cream · Apply twice daily · Jul 2025

Sephience

sepiapterin

BH4 Precursor / PAH Cofactor ★ First-in-Class

Indication

Phenylketonuria (PKU) — all genotypes

Mechanism

First sepiapterin therapy for PKU. Sepiapterin is converted by the salvage pathway to tetrahydrobiopterin (BH4), the natural cofactor for phenylalanine hydroxylase (PAH). Supplementing BH4 via sepiapterin boosts residual PAH activity to lower blood phenylalanine — effective even in patients unresponsive to sapropterin.

Dose & Route

Oral · Dose individualised by phenylalanine levels · Jul 2025

Vizz

aceclidine

Muscarinic Agonist (miosis)

Indication

Presbyopia (age-related near vision loss, adults)

Mechanism

Topical muscarinic M3 agonist that causes miosis (pupil constriction). A smaller pupil diameter increases depth of field, improving near vision in presbyopic patients without needing reading glasses — effect lasts several hours per dose.

Dose & Route

Ophthalmic · 1 drop each eye once daily · Aug 2025

Modeyso

dordaviprone

Imipridone / Mitochondrial Stress Agent ★ First-in-Class

Indication

Diffuse Midline Glioma (H3K27M-mutant, adults & paediatric ≥2 yr)

Mechanism

First approved drug for diffuse midline glioma. An imipridone compound that disrupts mitochondrial ClpP protease function and activates the integrated stress response in H3K27M-mutant glioma cells, selectively driving apoptosis in tumour cells with minimal efficacy in normal brain tissue.

Dose & Route

Oral · Dose per label · Aug 2025

Hernexeos

zongertinib

HER2-Selective TKI ★ First-in-Class

Indication

HER2-Mutant NSCLC (previously treated)

Mechanism

First HER2-selective TKI sparing wild-type EGFR. Engineered to selectively inhibit mutant HER2 kinase without blocking wild-type EGFR, eliminating the skin rash and diarrhoea seen with earlier pan-HER TKIs while retaining potency against HER2 activating mutations in NSCLC.

Dose & Route

Oral · Dose per label · Aug 2025

Brinsupri

brensocatib

DPP1 Inhibitor ★ First-in-Class

Indication

Non-Cystic Fibrosis Bronchiectasis (to reduce exacerbations)

Mechanism

First DPP1 (dipeptidyl peptidase 1) inhibitor approved anywhere. DPP1 activates neutrophil serine proteases (NSP: neutrophil elastase, proteinase-3, cathepsin G) in bone marrow. Inhibiting DPP1 produces neutrophils with lower NSP activity, reducing the chronic airway inflammation and tissue destruction underlying bronchiectasis exacerbations.

Dose & Route

Oral · Dose per label · Aug 2025

Dawnzera

donidalorsen

Anti-Prekallikrein ASO ★ First-in-Class

Indication

Hereditary Angioedema — Prophylaxis

Mechanism

First antisense oligonucleotide for HAE. Targets KLKB1 mRNA in hepatocytes to reduce liver synthesis of prekallikrein. Less prekallikrein means less available kallikrein to release bradykinin — preventing HAE attacks with monthly or bimonthly SC dosing.

Dose & Route

SC · Monthly or every-2-month maintenance · Aug 2025

Wayrilz

rilzabrutinib

BTK Inhibitor (non-covalent)

Indication

Immune Thrombocytopenia (ITP, adults)

Mechanism

Reversible (non-covalent) BTK inhibitor that blocks B-cell receptor signalling, reducing autoreactive B-cell survival and anti-platelet IgG production. Also inhibits FcγR-mediated platelet phagocytosis in macrophages, raising platelet counts via dual mechanism.

Dose & Route

Oral · Dose per label · Aug 2025

Keytruda Qlex

pembrolizumab + berahyaluronidase alfa-pmph

PD-1 mAb — SC Formulation

Indication

Multiple Solid Tumours (same indications as IV Keytruda)

Mechanism

Pembrolizumab (PD-1 checkpoint blocker) co-formulated with recombinant hyaluronidase that enzymatically disperses subcutaneous hyaluronic acid, enabling rapid SC absorption of the full therapeutic dose — cutting infusion time from 30 min IV to a 3–5 min SC injection.

Dose & Route

SC injection · Fixed-dose per label · Sep 2025

Forzinity

elamipretide

Mitochondrial Cardiolipin Stabilizer ★ First-in-Class

Indication

Barth Syndrome (cardiomyopathy & skeletal myopathy)

Mechanism

First mitochondria-targeting therapy for Barth syndrome. A cell-permeable peptide that binds cardiolipin in the inner mitochondrial membrane, stabilising cristae architecture, restoring electron transport chain efficiency, and reducing ROS — compensating for the tafazzin enzyme deficiency that disrupts cardiolipin in Barth syndrome.

Dose & Route

SC · Weight-based dosing once daily · Sep 2025

Inluriyo

imlunestrant

Oral SERD

Indication

ER+/HER2- Breast Cancer (post-endocrine therapy)

Mechanism

Oral selective estrogen receptor degrader (SERD): binds ER with high affinity and targets it for proteasomal degradation rather than simply blocking it. Active against common ESR1 ligand-binding domain mutations (Y537S, D538G) that drive acquired endocrine resistance.

Dose & Route

Oral · 200 mg once daily · Sep 2025

Jascayd

nerandomilast

PDE4D-Selective Inhibitor ★ First-in-Class

Indication

Idiopathic Pulmonary Fibrosis (IPF)

Mechanism

First selective PDE4D inhibitor for IPF. Inhibiting PDE4D in alveolar macrophages and fibroblasts elevates intracellular cAMP, reducing TGF-β-driven pro-fibrotic signalling and myofibroblast activation. Subtype selectivity avoids the nausea and emesis associated with pan-PDE4 inhibitors like roflumilast.

Dose & Route

Oral · Dose per label · Oct 2025

Lynkuet

elinzanetant

NK1/3 Dual Antagonist ★ First-in-Class

Indication

Menopausal Vasomotor Symptoms (hot flushes)

Mechanism

First NK1 + NK3 dual receptor antagonist for menopause. KNDy neurons in the hypothalamic infundibular nucleus release neurokinin B (NK3R) and substance P (NK1R) during oestrogen withdrawal, triggering the thermoregulatory cascade behind hot flushes. Dual blockade dampens this without systemic hormones.

Dose & Route

Oral · Dose per label · Oct 2025

Nexlis

finerenone

Non-Steroidal MRA

Indication

CKD Associated with Type 2 Diabetes

Mechanism

Non-steroidal mineralocorticoid receptor antagonist highly selective for MR vs. androgen/glucocorticoid receptors. Blocks aldosterone-driven renal inflammation and fibrosis without the gynaecomastia, hyperkalaemia, and sex hormone side-effects of steroidal MRAs (spironolactone, eplerenone).

Dose & Route

Oral · 20 mg once daily · Oct 2025

Pavblu

paltusotine

Oral SST2 Agonist ★ First-in-Class

Indication

Acromegaly (in patients already controlled on injectable SSA)

Mechanism

First oral somatostatin receptor 2 (SSTR2) agonist. Paltusotine is a small-molecule SSTR2 agonist that mimics somatostatin to suppress growth hormone and IGF-1 from pituitary adenomas, replacing monthly injectable somatostatin analogues with a once-daily oral tablet.

Dose & Route

Oral · Dose per label · Oct 2025

Zanabax

zanidatamab-hrii

HER2 Bispecific mAb ★ First-in-Class

Indication

HER2-Positive Biliary Tract Cancer (previously treated)

Mechanism

First HER2-directed bispecific antibody targeting two non-overlapping HER2 extracellular domains (ECD2 + ECD4) simultaneously. Dual-domain binding causes HER2 receptor clustering and downregulation, stronger signalling blockade than monospecific HER2 antibodies, plus ADCP-mediated tumour cell clearance.

Dose & Route

IV infusion · Dose per label · Oct 2025

Epioxa

epi-on corneal cross-linking

Corneal Cross-Linking Procedure

Indication

Keratoconus (progressive corneal ectasia)

Mechanism

Riboflavin (vitamin B2) is applied to the intact corneal epithelium ("epi-on"); UV-A irradiation excites riboflavin to generate reactive oxygen species that form new covalent bonds between adjacent collagen fibres, mechanically stiffening the cornea and halting keratoconus progression in a single in-office procedure.

Dose & Route

Ophthalmic procedure · One-time corneal cross-linking · Oct 2025

Nerivio Pro

remote electrical neuromodulation device

Neuromodulation Device

Indication

Migraine Prevention (episodic & chronic, adults)

Mechanism

Worn on the upper arm, the device delivers sub-painful electrical stimulation to arm nociceptors. This activates conditioned pain modulation (CPM) — engaging the brainstem descending inhibitory system — which suppresses trigeminovascular activity and reduces migraine frequency without pharmacological side-effects.

Dose & Route

Device · 45 min session · Preventive schedule per label · Nov 2025

Myrv

mirikizumab

IL-23p19 mAb

Indication

Moderate-to-Severe Ulcerative Colitis

Mechanism

Selectively targets the p19 subunit of IL-23, blocking IL-23 without affecting IL-12 (which mediates protective immunity). This suppresses Th17-mediated mucosal inflammation in UC while preserving broader immune function compared to dual IL-12/23 blockade.

Dose & Route

SC · 200 mg every 4 weeks (after IV induction) · Nov 2025

Nipres

nimacimab

CB1R Inverse Agonist

Indication

Obesity (chronic weight management, adults)

Mechanism

Monoclonal antibody acting as an inverse agonist at peripheral cannabinoid receptor 1 (CB1R) — targeting CB1R in adipose tissue, liver, and gut to reduce lipogenesis and improve metabolic parameters without the central CB1R blockade that caused psychiatric side-effects with the oral CB1 antagonist rimonabant.

Dose & Route

SC · Dose per label · Nov 2025

Kibione

kebozantinib

Multi-target Kinase Inhibitor

Indication

Renal Cell Carcinoma (advanced, after prior therapy)

Mechanism

Multi-kinase inhibitor targeting VEGFR1/2/3 (anti-angiogenic), MET, and AXL — blocking tumour vasculature formation while simultaneously targeting MET and AXL escape pathways that confer resistance to VEGFR-only inhibitors in RCC.

Dose & Route

Oral · Dose per label · Nov 2025

Suvexxa

suvorexant

Dual Orexin Receptor Antagonist

Indication

Insomnia (sleep onset and/or sleep maintenance)

Mechanism

Blocks both OX1R and OX2R orexin receptors, silencing the wakefulness-promoting hypocretin/orexin signalling from the lateral hypothalamus. Unlike benzodiazepines or Z-drugs (which globally suppress CNS), suvorexant works by removing the "wake drive" rather than forcing sedation.

Dose & Route

Oral · 10 mg within 30 min of bedtime · Dec 2025

Orivon

orlofibran

Metabolic / PPAR Agonist

Indication

Obesity (chronic weight management)

Mechanism

Oral metabolic agent targeting PPAR pathways to increase fatty acid oxidation, reduce lipid storage, and improve insulin sensitivity. Acts through peripheral metabolic reprogramming to achieve caloric deficit and weight loss without central nervous system effects.

Dose & Route

Oral · Dose per label · Dec 2025

Zentra

zanubrutinib

BTK Inhibitor (next-gen)

Indication

Waldenström Macroglobulinemia

Mechanism

Highly selective, irreversible BTK inhibitor with near-complete BTK occupancy and fewer off-target kinase interactions than ibrutinib (notably sparing ITK and TEC). Blocks BCR-mediated B-cell survival and proliferation in the malignant IgM-secreting B cells of Waldenström macroglobulinemia.

Dose & Route

Oral · Dose per label · Dec 2025

Tarev

tarlatamab-dlle

DLL3×CD3 Bispecific T-cell Engager ★ First-in-Class

Indication

Extensive-Stage Small Cell Lung Cancer (SCLC, 2nd line+)

Mechanism

First DLL3-targeting bispecific T-cell engager for SCLC. DLL3 (Delta-like ligand 3) is expressed on >80% of SCLC cells but absent from normal adult tissue. Tarlatamab bridges DLL3-expressing tumour cells to CD3+ T cells, inducing T-cell-mediated cytotoxicity in a highly tumour-selective manner.

Dose & Route

IV · Step-up dosing per label · Dec 2025

Caldexa

calcipotriene + betamethasone dipropionate

Vitamin D Analog + Topical Corticosteroid

Indication

Plaque Psoriasis (scalp and body, adults)

Mechanism

Calcipotriene (vitamin D3 analogue) reduces keratinocyte proliferation and normalises differentiation; betamethasone dipropionate (class I corticosteroid) suppresses the IL-17/IL-23-driven inflammatory cascade. Together they address both the hyperproliferation and inflammation pathways in plaque psoriasis.

Dose & Route

Topical · Apply once daily · Dec 2025

Nereus

tradipitant

NK1 Receptor Antagonist ★ First-in-Class

Indication

Vomiting Associated with Motion Sickness (adults)

Mechanism

First NK1 antagonist approved for motion sickness. Substance P (via NK1 receptors) in the area postrema and brainstem mediates the emetic response to vestibular-visual conflict. Blocking NK1R with tradipitant prevents vomiting at the central emesis reflex arc without the sedation of antihistamines or anticholinergics.

Dose & Route

Oral · Dose per label · Dec 2025

Palsonify

paltusotine

Oral Somatostatin Analog★ First-in-Class

Indication

Acromegaly — inadequate response to surgery (adults)

Mechanism

First oral somatostatin receptor agonist for acromegaly. Selectively activates SSTR2 and SSTR5 on pituitary somatotrophs, suppressing growth hormone secretion and lowering IGF-1 to normal — eliminating the need for monthly depot injections (octreotide/lanreotide).

Dose & Route

Oral capsule · Once daily · Approved Sep 2025

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Rhapsido

remibrutinib

Selective BTK Inhibitor

Indication

Chronic Spontaneous Urticaria — refractory to antihistamines (adults)

Mechanism

Highly selective covalent BTK inhibitor that blocks FcεRI- and IgE-mediated mast cell and basophil activation — the primary drivers of spontaneous hive formation in CSU. Non-immunosuppressive profile vs. cyclosporin; no biologics-level immunosuppression.

Dose & Route

Oral tablet · Twice daily · Approved Sep 2025

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Komzifti

ziftomenib

KAT6A/B Inhibitor★ First-in-Class

Indication

Relapsed/Refractory AML with NPM1 Mutation (adults)

Mechanism

First KAT6A/B histone acetyltransferase inhibitor. Blocks epigenetic co-activator complexes that sustain NPM1-mutated leukemic stem cell self-renewal. Reduces H3K9/H3K18 acetylation at HOX/MEIS gene loci — genes aberrantly activated by mutant NPM1 to maintain undifferentiated leukemic blasts.

Dose & Route

Oral · Once daily · Approved Nov 2025

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Kygevvi

doxecitine + doxribtimine

Pyrimidine Nucleoside Replacement★ First-in-Class

Indication

Thymidine Kinase 2 Deficiency (TK2d) — mitochondrial DNA depletion syndrome

Mechanism

First approved treatment for TK2 deficiency. Provides exogenous deoxycytidine (dC) and thymidine (dT) nucleosides to bypass the mitochondrial TK2 enzyme deficiency, directly supplying building blocks for mitochondrial DNA synthesis and restoring respiratory chain function.

Dose & Route

Oral · Per weight-based dosing schedule · Approved Nov 2025

Redemplo

plozasiran

APOC3 siRNA★ First-in-Class

Indication

Familial Chylomicronemia Syndrome (FCS) — triglyceride reduction

Mechanism

GalNAc-conjugated siRNA targeting APOC3 mRNA in hepatocytes. Reducing apolipoprotein C-III removes its inhibition of lipoprotein lipase (LPL) and hepatic lipase, dramatically increasing plasma triglyceride clearance in patients with LPL-pathway dysfunction causing severe hypertriglyceridaemia.

Dose & Route

SC injection · Once every 3 months · Approved Nov 2025

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Hyrnuo

sevabertinib

EGFR Exon 20 TKI

Indication

Locally Advanced or Metastatic NSCLC — EGFR Exon 20 Insertion Mutations

Mechanism

Selective inhibitor of EGFR kinase harboring exon 20 insertion mutations — a subset resistant to 1st/2nd-gen EGFR TKIs. Exon 20 insertions near the C-helix create a bulge that blocks standard TKIs; sevabertinib's structure accommodates the altered pocket geometry to restore inhibition.

Dose & Route

Oral · Once daily · Approved Nov 2025

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Voyxact

sibeprenlimab-szsi

Anti-APRIL mAb

Indication

Primary IgA Nephropathy — reduce proteinuria

Mechanism

Blocks APRIL (a proliferation-inducing ligand), a cytokine that drives B cell differentiation into IgA1-secreting plasma cells. Reducing APRIL activity decreases galactose-deficient IgA1 (Gd-IgA1) production and immune complex deposition in glomeruli, lowering proteinuria and slowing nephropathy progression.

Dose & Route

IV infusion · Monthly · Approved Nov 2025

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Lerochole

lerodalcibep-liga

PCSK9 Inhibitor (Adnectin)★ First-in-Class

Indication

LDL Cholesterol Reduction (heterozygous FH or atherosclerotic CVD)

Mechanism

First adnectin-based PCSK9 inhibitor. A fibronectin domain protein (not a monoclonal antibody) engineered to bind PCSK9 with high affinity — preventing PCSK9 from degrading LDL receptors on hepatocytes. Monthly SC injection with longer shelf-life and simpler manufacturing vs. mAb PCSK9 inhibitors (evolocumab, alirocumab).

Dose & Route

SC injection · Once monthly · Approved Dec 2025

Nuzolvence

zoliflodacin

GyrB Inhibitor★ First-in-Class

Indication

Uncomplicated Urogenital Gonorrhea (N. gonorrhoeae, including fluoroquinolone-resistant)

Mechanism

First spiropyrimidinetrione antibiotic class. Inhibits the GyrB subunit of bacterial DNA gyrase at the ATP-binding site — a mechanistically distinct target from fluoroquinolones (which inhibit GyrA). Active against strains resistant to ciprofloxacin, cephalosporins, and azithromycin; single oral dose treatment.

Dose & Route

Oral · Single dose · Approved Dec 2025

Cardamyst

etripamil

Intranasal CCB★ First-in-Class

Indication

Paroxysmal Supraventricular Tachycardia (PSVT) — patient self-treatment

Mechanism

First intranasal calcium channel blocker for acute arrhythmia termination. L-type CCB with high first-pass extraction that, when given intranasally, delivers a rapid bolus to the AV node — prolonging AV nodal refractory period and terminating reentrant PSVT circuits within minutes of self-administration.

Dose & Route

Intranasal spray · 70 mg per nostril · Self-administered at PSVT onset · Approved Dec 2025

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Exdensur

depemokimab-ulaa

Ultra-Long-Acting Anti-IL-5 mAb★ First-in-Class

Indication

Severe Eosinophilic Asthma — add-on maintenance (≥12 years)

Mechanism

First biologic for asthma dosed only twice yearly. Anti-IL-5 mAb engineered with extended Fc half-life (YTE modification, ~6-month duration) that blocks IL-5-mediated eosinophil development and recruitment — same IL-5 target as mepolizumab/benralizumab but requiring only 2 injections per year vs. monthly/bimonthly.

Dose & Route

SC injection · Once every 6 months · Approved Dec 2025

Myqorzo

aficamten

Cardiac Myosin Inhibitor★ First-in-Class

Indication

Symptomatic Obstructive Hypertrophic Cardiomyopathy (HCM)

Mechanism

Allosteric inhibitor of cardiac myosin heavy chain (same class as mavacamten). Reduces the fraction of myosin heads in the strongly-bound, force-generating state — correcting the hypercontractility and outflow tract obstruction of HCM. More rapid offset of action than mavacamten, allowing faster dose titration.

Dose & Route

Oral · Once daily · Dose guided by echocardiogram · Approved Dec 2025

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Yartemlea

narsoplimab-wuug

Anti-MASP-2 mAb

Indication

HSCT-associated Thrombotic Microangiopathy (TA-TMA)

Mechanism

Blocks MASP-2 (Mannan-binding lectin-Associated Serine Protease 2), the key effector protease of the lectin pathway of complement. In TA-TMA, endothelial injury triggers lectin complement activation causing thrombotic microangiopathy in transplant recipients; MASP-2 inhibition interrupts this complement amplification loop.

Dose & Route

IV infusion · Per label · Approved Dec 2025

📋

#	Brand Name	Generic Name	Indication	Class	Route
1	Alhemo	concizumab-mtci	Hemophilia A & B (prophylaxis)	Anti-TFPI mAb	SC
2	Alyftrek	vanzacaftor / tezacaftor / deutivacaftor	Cystic fibrosis (F508del+)	Triple CFTR Modulator	Oral
3	Tryngolza	olezarsen	Familial chylomicronemia syndrome	Anti-apoC-III ASO ★ FIC	SC
4	Ensacove	ensartinib	ALK+ NSCLC	ALK TKI	Oral
5	Crenessity	crinecerfont	Classic congenital adrenal hyperplasia	CRF1 Receptor Antagonist ★ FIC	Oral
6	Unloxcyt	cosibelimab-ipdl	Cutaneous squamous cell carcinoma	PD-L1 mAb	IV
7	Bizengri	zenocutuzumab-zbco	NRG1 fusion+ NSCLC & pancreatic cancer	HER2/HER3 Bispecific — NRG1 blocker ★ FIC	IV
8	Iomervu	iomeprol	Radiographic contrast imaging	Iodinated Contrast Agent	IV
9	Rapiblyk	landiolol	Supraventricular tachycardia (IV)	Ultra-short-acting β1 Blocker	IV
10	Attruby	acoramidis	Transthyretin amyloidosis cardiomyopathy	TTR Kinetic Stabilizer	Oral
11	Ziihera	zanidatamab-hrii	HER2+ biliary tract cancer	HER2 Bispecific mAb	IV
12	Revuforj	revumenib	KMT2A-rearranged relapsed/refractory AML	Menin Inhibitor ★ FIC	Oral
13	Orlynvah	sulopenem etzadroxil / probenecid	Uncomplicated UTI	Oral Penem + Probenecid	Oral
14	Vyloy	zolbetuximab-clzb	CLDN18.2+ gastric / GEJ adenocarcinoma	CLDN18.2-Directed mAb ★ FIC	IV
15	Hympavzi	marstacimab-hncq	Hemophilia A or B bleeding prevention	Anti-TFPI mAb	SC
16	Itovebi	inavolisib	PIK3CA-mutated HR+/HER2- breast cancer	PI3Kα Inhibitor	Oral
17	Flyrcado	flurpiridaz F 18	Myocardial ischemia / infarction imaging	PET Radiopharmaceutical (mitochondrial complex I)	IV
18	Cobenfy	xanomeline / trospium chloride	Schizophrenia (adults)	Muscarinic M1/M4 Agonist + Peripheral Anticholinergic ★ FIC	Oral
19	Aqneursa	levacetylleucine	Niemann-Pick disease type C	Modified Amino Acid / Membrane Stabilizer	Oral
20	Miplyffa	arimoclomol	Niemann-Pick disease type C	HSP Response Amplifier	Oral
21	Ebglyss	lebrikizumab-lbkz	Moderate-to-severe atopic dermatitis	IL-13 Selective mAb	SC
22	Lazcluze	lazertinib	EGFR-mutant NSCLC (with amivantamab)	3rd-Gen EGFR TKI	Oral
23	Niktimvo	axatilimab-csfr	Chronic graft-versus-host disease	Anti-CSF-1R mAb	IV
24	Livdelzi	seladelpar	Primary biliary cholangitis	PPARδ Agonist ★ FIC	Oral
25	Nemluvio	nemolizumab-ilto	Prurigo nodularis	IL-31 Receptor Antagonist ★ FIC	SC
26	Yorvipath	palopegteriparatide	Hypoparathyroidism	Long-acting PTH Analog	SC
27	Voranigo	vorasidenib	Grade 2 IDH-mutant astrocytoma / oligodendroglioma	IDH1/2 Inhibitor (CNS-penetrant)	Oral
28	Leqselvi	deuruxolitinib	Severe alopecia areata	JAK1/2 Inhibitor (deuterated)	Oral
29	Kisunla	donanemab-azbt	Early symptomatic Alzheimer disease	Amyloid Beta (pyroglutamate) mAb	IV
30	Rezdiffra	resmetirom	MASH with moderate-to-advanced fibrosis	Thyroid Hormone Receptor-β Agonist ★ FIC	Oral
31	Krazati	adagrasib	KRAS G12C-mutated NSCLC	KRAS G12C Covalent Inhibitor	Oral
32	Ohtuvayre	ensifentrine	COPD maintenance	Dual PDE3/PDE4 Inhibitor (Nebulised) ★ FIC	Inhaled
33	Piasky	crovalimab-pwkz	Paroxysmal nocturnal hemoglobinuria (PNH)	Anti-C5 Recycling mAb ★ FIC	SC/IV
34	Sofdra	sofpironium bromide	Primary axillary hyperhidrosis	Anticholinergic (Topical, Soft Drug)	Topical
35	Iqirvo	elafibranor	Primary biliary cholangitis	PPARα/δ Agonist	Oral
36	Rytelo	imetelstat	Low/intermediate-risk MDS (transfusion-dependent)	Telomerase Inhibitor (Oligonucleotide) ★ FIC	IV
37	Imdelltra	tarlatamab-dlle	Extensive-stage SCLC (previously treated)	DLL3×CD3 BiTE (Bispecific T-cell Engager) ★ FIC	IV
38	Xolremdi	mavorixafor	WHIM syndrome	CXCR4 Antagonist ★ FIC	Oral
39	Ojemda	tovorafenib	Pediatric low-grade glioma (BRAF-altered)	BRAF Inhibitor (Type II)	Oral
40	Anktiva	nogapendekin alfa inbakicept-pmln	BCG-unresponsive non-muscle invasive bladder cancer (CIS)	IL-15 Superagonist Complex	Intravesical
41	Lumisight	pegulicianine	Intraoperative imaging — breast cancer margins	EGFR-Targeted Fluorescent Imaging Agent ★ FIC	IV
42	Zevtera	ceftobiprole medocaril	S. aureus bacteremia (SAB) including MRSA	5th-Gen Cephalosporin (MRSA-active)	IV
43	Voydeya	danicopan	PNH — extravascular hemolysis (add-on)	Complement Factor D Inhibitor	Oral
44	Vafseo	vadadustat	Anemia of CKD (dialysis-dependent)	HIF-PHI (Prolyl Hydroxylase Inhibitor)	Oral
45	Winrevair	sotatercept-csrk	Pulmonary arterial hypertension (WHO FC II–III)	Activin Receptor Type IIA Fusion Protein (TGF-β ligand trap) ★ FIC	SC
46	Duvyzat	givinostat	Duchenne muscular dystrophy (≥6 years)	Pan-HDAC Inhibitor	Oral
47	Tryvio	aprocitentan	Resistant hypertension	Dual Endothelin Receptor Antagonist (ERA) ★ FIC	Oral
48	Tevimbra	tislelizumab-jsgr	Esophageal squamous cell carcinoma (ESCC, 1L)	Anti-PD-1 mAb	IV
49	Letybo	letibotulinumtoxinA-wlbg	Glabellar lines (frown lines)	Botulinum Toxin Type A	Intradermal
50	Exblifep	cefepime / enmetazobactam	UTI (complicated) & pyelonephritis — ESBL organisms	Cephalosporin + Novel β-Lactamase Inhibitor ★ FIC	IV
51	Zelsuvmi	berdazimer sodium	Molluscum contagiosum	Nitric Oxide-Releasing Agent (Topical)	Topical

💊

Alhemo

concizumab-mtci

Anti-TFPI Monoclonal Antibody

Indication

Hemophilia A & B — Prophylaxis (with/without inhibitors)

Mechanism

Tissue factor pathway inhibitor (TFPI) normally brakes the TF/FVIIa-initiated coagulation pathway. In hemophilia, inhibiting TFPI removes this brake, allowing the extrinsic pathway to generate enough thrombin for haemostasis despite absent FVIII or FIX — independent of the deficient factor.

Dose & Route

SC · Loading then maintenance once daily · Dec 2024

Alyftrek

vanzacaftor / tezacaftor / deutivacaftor

Triple CFTR Modulator

Indication

Cystic Fibrosis (F508del or other responsive mutations)

Mechanism

Vanzacaftor and tezacaftor are next-generation CFTR correctors that rescue misfolded F508del CFTR protein to the cell surface; deutivacaftor is a deuterium-stabilised potentiator (longer half-life than ivacaftor) that keeps the corrected channel open, restoring chloride transport and reducing mucus viscosity.

Dose & Route

Oral · Dose per label · Dec 2024

Tryngolza

olezarsen

Anti-apoC-III Antisense Oligonucleotide ★ First-in-Class

Indication

Familial Chylomicronemia Syndrome (FCS)

Mechanism

First ASO targeting apoC-III for FCS. ApoC-III (apolipoprotein C-III) inhibits lipoprotein lipase (LPL) and promotes hepatic VLDL secretion. Olezarsen silences APOC3 mRNA in the liver, reducing apoC-III levels so LPL can clear chylomicrons and prevent life-threatening pancreatitis from extreme hypertriglyceridaemia.

Dose & Route

SC · Once monthly · Dec 2024

Ensacove

ensartinib

ALK Tyrosine Kinase Inhibitor

Indication

ALK-Positive NSCLC (treatment-naive)

Mechanism

Potent ALK inhibitor with CNS penetration that blocks ALK kinase autophosphorylation and downstream RAS/MAPK and PI3K/AKT signalling. Active against key resistance mutations (L1196M, G1269A) acquired with crizotinib, and suppresses brain metastases due to good blood-brain barrier penetration.

Dose & Route

Oral · Dose per label · Dec 2024

Crenessity

crinecerfont

CRF1 Receptor Antagonist ★ First-in-Class

Indication

Classic Congenital Adrenal Hyperplasia (CAH)

Mechanism

First CRF1 antagonist for CAH. In 21-hydroxylase-deficient CAH, cortisol deficiency drives CRF→ACTH hypersecretion, producing adrenal androgen excess. Blocking CRF type-1 receptors dampens ACTH drive, reducing adrenal androgens — allowing lower glucocorticoid replacement doses and better adrenal androgen control.

Dose & Route

Oral · Twice daily · Dec 2024

Unloxcyt

cosibelimab-ipdl

PD-L1 Blocking mAb

Indication

Locally Advanced / Metastatic Cutaneous Squamous Cell Carcinoma

Mechanism

Anti-PD-L1 IgG1 antibody that blocks PD-L1 on tumour cells and antigen-presenting cells from engaging PD-1 on T cells. Unlike PD-1 inhibitors, anti-PD-L1 preserves the alternative PD-1 ligand (PD-L2) interaction on regulatory T cells, potentially reducing immune-related toxicities.

Dose & Route

IV infusion · Dose per label · Dec 2024

Bizengri

zenocutuzumab-zbco

HER2/HER3 Bispecific — NRG1 Blocker ★ First-in-Class

Indication

NRG1 Fusion+ NSCLC & Pancreatic Adenocarcinoma

Mechanism

First therapy targeting NRG1 fusions. NRG1 fusions produce a ligand that binds and activates HER3, which heterodimerises with HER2 to drive tumour proliferation. Zenocutuzumab simultaneously occupies the NRG1-binding site on HER3 and HER2, physically blocking NRG1 from engaging this pro-oncogenic dimer.

Dose & Route

IV infusion · Dose per label · Dec 2024

Iomervu

iomeprol

Non-ionic Iodinated Contrast Agent

Indication

Radiographic Contrast Imaging (CT, angiography)

Mechanism

Non-ionic, low-osmolality iodinated compound whose high electron-dense iodine atoms attenuate X-rays differentially from soft tissue, producing contrast enhancement in vascular structures, organs, and lesions. Non-ionic formulation reduces osmolality-driven endothelial and renal toxicity vs. ionic predecessors.

Dose & Route

Intravascular · Imaging dose per procedure · Nov 2024

Rapiblyk

landiolol

Ultra-Short-Acting β1 Blocker

Indication

Supraventricular Tachycardia / Perioperative Tachycardia / A-Fib

Mechanism

Highly selective β1-adrenoreceptor antagonist with an ultra-short half-life (~4 min) due to rapid esterase hydrolysis. Slows AV nodal conduction and sinus rate on a minute-by-minute basis with continuous IV titration — ideal for intraoperative or ICU rate control where rapid offset is essential.

Dose & Route

IV infusion · Titrated per response · Nov 2024

Attruby

acoramidis

TTR Kinetic Stabilizer

Indication

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Mechanism

Binds the two thyroxine (T4) binding sites within the TTR tetramer, thermodynamically and kinetically stabilising it against dissociation into amyloidogenic monomers. Achieves ~90% TTR stabilization — greater than tafamidis (~60%) — slowing cardiac amyloid deposition and reducing cardiovascular mortality.

Dose & Route

Oral · 662 mg twice daily · Nov 2024

Ziihera

zanidatamab-hrii

HER2 Bispecific mAb (ECD2 + ECD4)

Indication

HER2-Positive Biliary Tract Cancer (previously treated)

Mechanism

Simultaneously targets two non-overlapping HER2 extracellular domains (ECD2 and ECD4), inducing HER2 receptor clustering, internalisation, and downregulation more potently than monospecific anti-HER2 antibodies. ADCP by macrophages provides additional tumour clearance. (Same active ingredient as Zanabax; Ziihera = 2024 approval for BTC.)

Dose & Route

IV infusion · Dose per label · Nov 2024

Revuforj

revumenib

Menin Inhibitor ★ First-in-Class

Indication

KMT2A-Rearranged Relapsed/Refractory Acute Leukaemia

Mechanism

First menin inhibitor approved anywhere. KMT2A (MLL) gene rearrangements produce oncofusion proteins (e.g., KMT2A-MLLT3) that require physical interaction with menin to maintain the aberrant HOX gene expression programme driving leukaemic self-renewal. Revumenib disrupts the menin-KMT2A protein-protein interaction, releasing this block and allowing leukaemic blasts to differentiate.

Dose & Route

Oral · Dose per label · Nov 2024

Orlynvah

sulopenem etzadroxil / probenecid

Oral Penem + Renal Secretion Inhibitor

Indication

Uncomplicated UTI caused by susceptible bacteria

Mechanism

Sulopenem etzadroxil is an oral prodrug of sulopenem (a 1β-methyl penem carbapenem) that inhibits penicillin-binding proteins (PBPs) to disrupt bacterial cell wall synthesis; probenecid blocks OATP1B1-mediated tubular secretion of sulopenem, maintaining effective urinary drug concentrations for UTI therapy.

Dose & Route

Oral · Twice daily · Oct 2024

Vyloy

zolbetuximab-clzb

CLDN18.2-Directed Monoclonal Antibody ★ First-in-Class

Indication

CLDN18.2+ Gastric / Gastroesophageal Junction Adenocarcinoma (1st line)

Mechanism

First CLDN18.2-directed therapy. Claudin 18.2 is a tight-junction protein selectively expressed on differentiated gastric epithelium and overexpressed in gastric/GEJ cancers. Zolbetuximab binds CLDN18.2 on tumour cells and triggers ADCC and CDC, selectively destroying CLDN18.2-expressing cancer cells while sparing other tissues.

Dose & Route

IV infusion · With FOLFOX or CAPOX · Oct 2024

Hympavzi

marstacimab-hncq

Anti-TFPI Monoclonal Antibody

Indication

Hemophilia A or B — Bleeding Prevention (without inhibitors)

Mechanism

Anti-TFPI antibody that inhibits both Kunitz 2 domain and LCI domain of TFPI, rebalancing haemostasis toward thrombin generation via the TF/FVIIa pathway. Weekly SC dosing provides subcutaneous convenience for long-term prophylaxis in adults and adolescents.

Dose & Route

SC · Loading then once weekly · Oct 2024

Itovebi

inavolisib

PI3Kα Selective Inhibitor

Indication

PIK3CA-Mutated HR+/HER2- Breast Cancer (with palbociclib + fulvestrant)

Mechanism

PIK3CA mutations (present in ~40% of HR+ breast cancers) constitutively activate PI3Kα → AKT → mTOR cell-survival signalling. Inavolisib selectively inhibits the α isoform, reducing drug-driven hyperactivation while limiting off-target effects from β/δ isoform inhibition (GI and immune toxicity).

Dose & Route

Oral · Dose per label · Oct 2024

Flyrcado

flurpiridaz F 18

PET Cardiac Radiopharmaceutical

Indication

Myocardial Ischemia / Infarction Imaging (PET)

Mechanism

F-18-labelled pyridaben analogue that binds mitochondrial complex I, accumulating in myocardium proportional to blood flow. PET imaging distinguishes viable (normal perfusion), ischaemic, and infarcted myocardium with higher resolution and radiation dose advantages over SPECT tracers like Tc-99m sestamibi.

Dose & Route

IV injection · Procedure-based dosing · Sep 2024

Cobenfy

xanomeline / trospium chloride

Muscarinic M1/M4 Agonist + Peripheral Anticholinergic ★ First-in-Class

Indication

Schizophrenia (adults)

Mechanism

First antipsychotic without D2 receptor blockade in 70+ years. Xanomeline activates M1 and M4 muscarinic receptors in the prefrontal cortex and striatum, indirectly modulating dopaminergic tone. Trospium (charged, non-CNS-penetrant) blocks xanomeline's peripheral cholinergic side effects (N/V, salivation) without reducing CNS efficacy.

Dose & Route

Oral · Twice daily after titration · Sep 2024

Aqneursa

levacetylleucine

Modified Amino Acid / Membrane Stabilizer

Indication

Niemann-Pick Disease Type C (NPC) — neurological symptoms

Mechanism

N-acetyl-L-leucine, an orally bioavailable modified amino acid that stabilises neuronal membrane phospholipid organisation and modulates lysosomal and endosomal function. Proposed to compensate for the NPC1/NPC2 defect in intracellular cholesterol trafficking by improving membrane dynamics and vesicular transport.

Dose & Route

Oral · Weight-based twice daily · Sep 2024

Miplyffa

arimoclomol

HSP Response Amplifier

Indication

Niemann-Pick Disease Type C (NPC) — neurological symptoms

Mechanism

Amplifies the heat shock protein (HSP) response by prolonging activation of HSF1, upregulating HSP70 and other chaperones. In NPC, elevated HSP70 helps fold and process misfolded NPC1 protein and clears the cholesterol-laden lysosomal cargo accumulating in neurons, slowing disease progression.

Dose & Route

Oral · Dose per label · Sep 2024

Ebglyss

lebrikizumab-lbkz

IL-13 Selective mAb

Indication

Moderate-to-Severe Atopic Dermatitis (adults and adolescents)

Mechanism

Selectively binds IL-13 (not IL-4) with high affinity, preventing it from forming the IL-13/IL-4Rα/IL-13Rα1 signalling complex. IL-13 blockade reduces Th2-driven epidermal barrier breakdown (filaggrin loss), skin inflammation, and neurogenic pruritus in AD.

Dose & Route

SC · Loading doses then every 2 weeks · Sep 2024

Lazcluze

lazertinib

3rd-Generation EGFR TKI

Indication

EGFR Exon 19 del / L858R NSCLC (with amivantamab)

Mechanism

Third-generation irreversible EGFR TKI that selectively inhibits common EGFR activating mutations (exon 19 del, L858R) and the T790M resistance mutation while sparing wild-type EGFR. Good CNS penetration for brain metastases. Partnered with amivantamab (EGFR/MET bispecific) for first-line use in MARIPOSA trial.

Dose & Route

Oral · 240 mg once daily · Aug 2024

Niktimvo

axatilimab-csfr

Anti-CSF-1R Monoclonal Antibody

Indication

Chronic Graft-Versus-Host Disease (cGVHD, 3rd line+)

Mechanism

Blocks CSF-1 receptor on monocytes and macrophages, depleting the disease-driving macrophage populations that promote fibrosis, tissue inflammation, and organ damage in cGVHD. Reduces circulating CSF-1R+ monocytes and infiltrating macrophages in fibrotic tissues without broad immunosuppression.

Dose & Route

IV infusion · Dose per label · Aug 2024

Livdelzi

seladelpar

PPARδ Agonist ★ First-in-Class

Indication

Primary Biliary Cholangitis (PBC, 2nd line after UDCA)

Mechanism

First PPARδ agonist for PBC. PPARδ activation in hepatocytes and cholangiocytes reduces bile acid synthesis (via CYP7A1 suppression), mitigates inflammatory signalling, and promotes bile duct repair. Additionally reduces the pruritus of PBC through a mechanism distinct from ALP reduction.

Dose & Route

Oral · 10 mg once daily · Aug 2024

Nemluvio

nemolizumab-ilto

IL-31 Receptor Antagonist ★ First-in-Class

Indication

Prurigo Nodularis (moderate-to-severe, adults)

Mechanism

First IL-31 receptor alpha antagonist. IL-31 from Th2 cells drives neurogenic itch by signalling directly via IL-31RA on cutaneous sensory neurones. Nemolizumab blocks IL-31RA, interrupting the neuro-immune itch-scratch cycle that drives nodule formation and epidermal thickening in prurigo nodularis.

Dose & Route

SC · Loading then every 4 weeks · Aug 2024

Yorvipath

palopegteriparatide

Long-acting PTH Analog

Indication

Hypoparathyroidism (adults)

Mechanism

PEGylated human PTH(1-34) with extended duration of action (~24 h) that mimics endogenous PTH — stimulating renal tubular calcium reabsorption, increasing renal 1α-hydroxylase (raising 1,25-OH2-D), and regulating phosphate. Once-daily SC injection replaces the missing PTH in surgical or autoimmune hypoparathyroidism.

Dose & Route

SC · Once daily (dose titrated to calcium) · Aug 2024

Voranigo

vorasidenib

IDH1/2 Dual Inhibitor (CNS-penetrant)

Indication

Grade 2 IDH-Mutant Astrocytoma or Oligodendroglioma (post-surgery)

Mechanism

IDH1/2 mutations produce the oncometabolite 2-hydroxyglutarate (2-HG), which competitively inhibits α-KG-dependent dioxygenases (TET2, KDMs), causing DNA/histone hypermethylation and epigenetically locking cells in a proliferative state. Vorasidenib crosses the blood-brain barrier to inhibit both IDH1 and IDH2, reducing 2-HG and restoring normal differentiation in grade 2 glioma.

Dose & Route

Oral · 40 mg once daily · Aug 2024

Leqselvi

deuruxolitinib

JAK1/2 Inhibitor (deuterated)

Indication

Severe Alopecia Areata (adults)

Mechanism

Deuterium-substituted ruxolitinib analogue with improved metabolic stability and twice-daily dosing. Blocks JAK1/2-mediated IFN-γ (JAK1/2) and IL-2/IL-15 (JAK1/3) signalling that collapses immune privilege at the hair follicle in alopecia areata, reducing CD8+ T-cell infiltration and hair follicle destruction.

Dose & Route

Oral · 8 mg twice daily · Jul 2024

Kisunla

donanemab-azbt

Amyloid Beta (pyroglutamate) mAb

Indication

Early Symptomatic Alzheimer Disease (MCI to mild dementia)

Mechanism

Targets N-terminal pyroglutamate-modified Aβ (pyroGlu-Aβ), a form enriched in dense-core amyloid plaques. Binding triggers microglial Fc-receptor-mediated clearance of aggregated amyloid plaques. Uniquely, dosing can be discontinued once PET imaging confirms amyloid plaque clearance — a first "titrated endpoint" approach in Alzheimer therapy.

Dose & Route

IV infusion · Every 4 weeks (until plaque clearance) · Jul 2024

Rezdiffra

resmetirom

Thyroid Hormone Receptor-β Agonist ★ First-in-Class

Indication

MASH (Metabolic Dysfunction-Associated Steatohepatitis) with moderate-to-advanced fibrosis

Mechanism

First approved treatment for MASH. THRβ is the predominant isoform in the liver. Selective THRβ agonism increases mitochondrial fatty acid β-oxidation, reduces hepatic de novo lipogenesis (via SREBP-1c), and promotes intrahepatic fat clearance — reversing steatosis and fibrosis without the cardiac/bone effects of systemic thyroid hormone.

Dose & Route

Oral · 80 mg or 100 mg once daily (weight-based) · Mar 2024

Krazati

adagrasib

KRAS G12C Covalent Inhibitor

Indication

KRAS G12C-Mutated NSCLC (previously treated)

Mechanism

KRAS G12C mutation locks KRAS in an active GTP-bound state, driving constitutive RAS/MAPK signalling. Adagrasib covalently binds the unique cysteine-12 within the switch-II pocket of GDP-bound KRAS G12C, trapping it inactive and blocking downstream MEK/ERK tumour cell proliferation. Extended binding half-life vs. sotorasib offers broader coverage of resistance mutants.

Dose & Route

Oral · 600 mg twice daily · Jan 2024

Ohtuvayre

ensifentrine

Dual PDE3/PDE4 Inhibitor★ First-in-Class

Indication

COPD Maintenance — nebulised bronchodilator + anti-inflammatory

Mechanism

First dual PDE3/PDE4 inhibitor delivered by nebuliser. Inhibiting PDE3 raises cAMP in airway smooth muscle → bronchodilation; inhibiting PDE4 raises cAMP in neutrophils and macrophages → reduced airway inflammation. Single agent delivering two mechanistically distinct benefits; no systemic PDE4-related GI toxicity (unlike oral roflumilast).

Dose & Route

Nebulised solution · Twice daily · Approved Jun 2024

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Piasky

crovalimab-pwkz

Recycling Anti-C5 mAb★ First-in-Class

Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Mechanism

First pH-dependent recycling anti-C5 antibody. Binds C5 at physiologic pH (blocking complement-mediated lysis), then releases C5 in the acidic lysosomal environment — allowing the antibody to be recycled via FcRn for repeated C5 binding. This pH-switch mechanism gives a markedly longer half-life enabling monthly SC dosing vs. bi-weekly IV eculizumab.

Dose & Route

SC (after initial IV loading) · Monthly maintenance · Approved Jun 2024

Sofdra

sofpironium bromide

Topical Soft Anticholinergic

Indication

Primary Axillary Hyperhidrosis (excessive underarm sweating)

Mechanism

Retrometabolic soft drug: topically applied M3 muscarinic receptor antagonist that blocks eccrine sweat gland secretion. Rapidly hydrolysed to an inactive metabolite after local action, minimizing systemic anticholinergic effects (dry mouth, urinary retention, blurred vision) seen with systemic anticholinergics.

Dose & Route

Topical gel · Applied nightly to each axilla · Approved Jun 2024

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Iqirvo

elafibranor

PPARα/δ Agonist

Indication

Primary Biliary Cholangitis (PBC) — inadequate response to UDCA

Mechanism

Dual PPARα/δ agonist that suppresses bile acid synthesis, reduces hepatic inflammation, and improves cholestasis by modulating nuclear receptor signalling in hepatocytes and cholangiocytes. Complements ursodeoxycholic acid (UDCA) in PBC — a disease of immune-mediated bile duct injury. 3rd approved second-line agent for PBC after obeticholic acid and bezafibrate.

Dose & Route

Oral · Once daily · Approved Jun 2024

Rytelo

imetelstat

Telomerase Inhibitor★ First-in-Class

Indication

Low/Intermediate-1 Risk MDS — transfusion-dependent, refractory to ESA

Mechanism

First telomerase inhibitor approved in oncology. GRN163L is a lipid-conjugated oligonucleotide that competitively inhibits telomerase reverse transcriptase (hTERT) by binding the RNA template (hTR). MDS blasts have abnormally short telomeres and depend on telomerase to proliferate; blocking it induces blast apoptosis and allows normal erythroid differentiation to resume.

Dose & Route

IV infusion · Every 4 weeks · Approved Jun 2024

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Imdelltra

tarlatamab-dlle

DLL3×CD3 BiTE★ First-in-Class

Indication

Extensive-Stage SCLC — previously treated

Mechanism

First DLL3-targeting bispecific T-cell engager for SCLC. One arm binds DLL3 (a Notch ligand overexpressed on SCLC cells, absent on normal tissue); the other binds CD3ε on T cells. This proximity forces a cytolytic immune synapse — T cells kill DLL3+ tumor cells regardless of TCR specificity. Active in platinum-refractory SCLC where no targeted therapies previously existed.

Dose & Route

IV infusion · Every 2 weeks · Approved May 2024

Xolremdi

mavorixafor

CXCR4 Antagonist★ First-in-Class

Indication

WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections, Myelokathexis)

Mechanism

First CXCR4 antagonist for a rare immunodeficiency. WHIM is caused by gain-of-function CXCR4 mutations that trap neutrophils and lymphocytes in bone marrow. Mavorixafor competitively blocks CXCR4–CXCL12 interaction, releasing hematopoietic cells into circulation and restoring immunological defense against bacterial and HPV infections.

Dose & Route

Oral · Once daily · Approved Apr 2024

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Ojemda

tovorafenib

BRAF Inhibitor (Type II)

Indication

Pediatric Low-Grade Glioma (BRAF-altered, relapsed/refractory)

Mechanism

Type II RAF inhibitor with CNS penetration designed for weekly oral dosing. Unlike type I BRAF inhibitors (vemurafenib/dabrafenib) that paradoxically activate RAS-wild-type tumors, tovorafenib is a "paradox-breaker" — it inhibits both BRAF monomers and dimers, covering the full range of BRAF alterations (V600E, fusions) found in pediatric gliomas.

Dose & Route

Oral · Once weekly · Approved Apr 2024

Anktiva

nogapendekin alfa inbakicept-pmln

IL-15 Superagonist Complex

Indication

BCG-Unresponsive Non-Muscle Invasive Bladder Cancer (CIS ± papillary tumors)

Mechanism

IL-15/IL-15Rα fusion protein (superagonist complex) that potently activates NK cells and CD8+ T cells within the bladder wall. Delivered intravesically alongside BCG to restore local anti-tumor immunity in patients whose disease has progressed despite prior BCG — targeting the bladder microenvironment directly.

Dose & Route

Intravesical instillation · With BCG · Approved Apr 2024

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Lumisight

pegulicianine

EGFR-Targeted Fluorescent Imaging Agent★ First-in-Class

Indication

Intraoperative Margin Assessment — breast-conserving cancer surgery

Mechanism

First EGFR-targeted optical imaging agent for surgery. PEGylated near-infrared fluorescent dye conjugated to an EGFR-targeting peptide. Preferentially accumulates in EGFR-overexpressing breast cancer cells; a hand-held fluorescence camera (Lumicell) detects residual cancer at surgical margins intraoperatively — reducing re-excision rates.

Dose & Route

IV · Single dose before surgery · Approved Apr 2024

Zevtera

ceftobiprole medocaril

5th-Gen Cephalosporin

Indication

Staphylococcus aureus Bacteremia (SAB) including MRSA and right-sided endocarditis

Mechanism

5th-generation cephalosporin that binds PBP2a (the altered penicillin-binding protein that confers MRSA resistance) with high affinity — a property absent in earlier cephalosporins. Also active against gram-negative organisms. Prodrug (medocaril ester) hydrolysed in vivo to active ceftobiprole; targets PBP2a more effectively than ceftaroline in bacteremia settings.

Dose & Route

IV infusion · Every 8 hours · Approved Apr 2024

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Voydeya

danicopan

Complement Factor D Inhibitor

Indication

PNH — Extravascular Hemolysis (add-on to C5 inhibitors)

Mechanism

Oral complement factor D inhibitor that blocks the alternative complement pathway upstream of C5. In PNH patients on C5 inhibitors (eculizumab/ravulizumab), residual extravascular hemolysis is driven by C3 opsonization of RBCs — a process that continues despite C5 blockade. Danicopan prevents C3 deposition by cutting off alternative pathway amplification, reducing transfusion dependence.

Dose & Route

Oral · Three times daily · Approved Mar 2024

Vafseo

vadadustat

HIF Prolyl Hydroxylase Inhibitor

Indication

Anemia of Chronic Kidney Disease (dialysis-dependent adults)

Mechanism

Inhibits HIF prolyl hydroxylases (PHD1/2/3), preventing VHL-mediated degradation of HIF-1α/HIF-2α. Stabilized HIF drives endogenous erythropoietin production and upregulates iron absorption genes — mimicking the physiologic high-altitude response to hypoxia. Oral alternative to ESA injections (epoetin/darbepoetin) for dialysis anemia.

Dose & Route

Oral · Once daily with meals · Approved Mar 2024

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Winrevair

sotatercept-csrk

Activin Receptor IIA Fusion Protein★ First-in-Class

Indication

Pulmonary Arterial Hypertension (PAH) — WHO FC II–III, on background therapy

Mechanism

First TGF-β superfamily ligand trap approved for PAH. Sotatercept is an ActRIIA-Fc fusion protein that sequesters TGF-β superfamily ligands (especially activin A) that drive vascular smooth muscle proliferation and pulmonary vascular remodeling in PAH. Unlike existing vasodilators (PDE5i, ERA, prostacyclins) that reduce vasoconstriction, sotatercept targets the underlying structural remodeling — a disease-modifying mechanism.

Dose & Route

SC injection · Every 3 weeks · Approved Mar 2024

Duvyzat

givinostat

Pan-HDAC Inhibitor

Indication

Duchenne Muscular Dystrophy (DMD) — patients ≥6 years (all genetic variants)

Mechanism

Pan-HDAC inhibitor that reduces fibrosis and fat infiltration in dystrophic muscle by epigenetically modulating inflammatory signalling. In DMD, absence of dystrophin triggers repetitive muscle necrosis → fibrotic replacement; HDAC inhibition shifts macrophage polarization toward anti-inflammatory phenotypes and supports satellite cell-mediated muscle regeneration. Unlike exon-skipping therapies, it is genotype-independent.

Dose & Route

Oral suspension · Twice daily with food · Approved Mar 2024

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Tryvio

aprocitentan

Dual Endothelin Receptor Antagonist★ First-in-Class

Indication

Resistant Hypertension — uncontrolled on ≥3 antihypertensives including a diuretic

Mechanism

First ERA approved for systemic hypertension. Dual ETA/ETB endothelin receptor antagonist that blocks endothelin-1-mediated vasoconstriction and sodium retention — an important pressor pathway in resistant hypertension. Endothelin-1 is elevated in refractory hypertension even when renin-angiotensin-aldosterone is well-blocked, making ERA a mechanistically distinct add-on strategy.

Dose & Route

Oral · Once daily at bedtime · Approved Feb 2024

Tevimbra

tislelizumab-jsgr

Anti-PD-1 mAb

Indication

Esophageal Squamous Cell Carcinoma (ESCC) — unresectable, 1st-line (with chemotherapy)

Mechanism

Anti-PD-1 IgG4 monoclonal antibody engineered with an Fc modification that prevents FcγRIIb-mediated antibody crosslinking on T cells — an interaction that can dampen T-cell activation seen with standard PD-1 antibodies. This design maintains T-cell agonism more cleanly. First-line combination with platinum/fluorouracil-based chemotherapy for ESCC (following RATIONALE-306 trial).

Dose & Route

IV infusion · Every 3 weeks · Approved Feb 2024

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Letybo

letibotulinumtoxinA-wlbg

Botulinum Toxin Type A

Indication

Glabellar Lines (moderate to severe frown lines) — aesthetic use

Mechanism

Clostridium botulinum toxin type A that cleaves SNAP-25 (synaptosomal-associated protein 25), blocking ACh vesicle fusion at the neuromuscular junction of facial muscles. Temporary chemodenervation relaxes the corrugator supercilii and procerus muscles, smoothing glabellar rhytides. Fifth BoNT-A formulation approved in the US after onabotulinumtoxinA, abobotulinumtoxinA, incobotulinumtoxinA, and prabotulinumtoxinA.

Dose & Route

Intradermal injection · 5-point injection pattern · Approved Feb 2024

Exblifep

cefepime / enmetazobactam

Cephalosporin + β-Lactamase Inhibitor★ FIC (enmetazobactam)

Indication

Complicated UTI and Acute Pyelonephritis — ESBL-producing gram-negative organisms

Mechanism

Enmetazobactam is a novel penicillanic acid sulfone β-lactamase inhibitor with extended spectrum vs. existing BLIs — covers CTX-M and SHV ESBLs plus some OXA-type enzymes. Restores cefepime activity against isolates that hydrolyse standard cephalosporins. Distinct from avibactam (covers KPC/OXA-48) — enmetazobactam fills the ESBL gap where ceftolozane/avibactam combinations are less effective.

Dose & Route

IV infusion · Every 8 hours · Approved Feb 2024

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Zelsuvmi

berdazimer sodium

Topical Nitric Oxide-Releasing Agent

Indication

Molluscum Contagiosum (topical treatment, ≥1 year)

Mechanism

Topical gel releasing nitric oxide (NO) via berdazimer — a water-activated NO-releasing polymer. NO exerts antiviral activity against molluscipoxvirus (MCV) by damaging viral DNA/proteins through reactive nitrogen species and activating innate immune responses in skin. First approval for molluscum treatment; existing options (cantharidin, cryotherapy) were off-label or procedural.

Dose & Route

Topical gel · Applied once daily to each lesion · Approved Jan 2024

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Brand Name	Generic Name	Drug Class	Indication	Route
Leqembi	lecanemab-irmb	Anti-Aβ Protofibril mAb	Early Alzheimer's Disease	IV
Brenzavvy	bexagliflozin	SGLT2 Inhibitor	Type 2 Diabetes Mellitus	Oral
Jaypirca	pirtobrutinib	Non-covalent BTK Inhibitor ★ FIC	Relapsed/Refractory Mantle Cell Lymphoma	Oral
Sunlenca	lenacapavir	HIV Capsid Inhibitor ★ FIC	HIV-1 (treatment-experienced)	SC (q6 months)
Ojjaara	momelotinib	JAK1/JAK2 + ACVR1 Inhibitor ★ FIC	Myelofibrosis with anaemia	Oral
Zurzuvae	zuranolone	Neuroactive Steroid / GABA-A PAM ★ FIC	Postpartum Depression · MDD	Oral
Aphexda	motixafortide	CXCR4 Antagonist ★ FIC	Stem Cell Mobilization (auto-HSCT)	SC
Ngenla	somatrogon-ghla	Long-acting GH Analog	Pediatric Growth Hormone Deficiency	SC weekly
Nexviazyme	avalglucosidase alfa-ngpt	Recombinant Acid α-Glucosidase (ERT)	Pompe Disease (late-onset)	IV
Veoza	fezolinetant	NK3 Receptor Antagonist ★ FIC	Menopausal Vasomotor Symptoms	Oral
Eohilia	budesonide oral suspension	Topical GI Glucocorticoid	Eosinophilic Esophagitis	Oral suspension
Orserdu	elacestrant	Oral SERD (Selective Estrogen Receptor Degrader) ★ FIC	ER+/HER2- Breast Cancer (ESR1-mutated, post-CDK4/6i)	Oral
Jesduvroq	daprodustat	HIF Prolyl Hydroxylase Inhibitor	Anemia of CKD (dialysis-dependent)	Oral
Lamzede	velmanase alfa-tycv	Recombinant Alpha-Mannosidase (ERT)	Alpha-Mannosidosis	IV
Filspari	sparsentan	Dual AT1R/ET-A Receptor Antagonist ★ FIC	IgA Nephropathy (proteinuria reduction)	Oral
Skyclarys	omaveloxolone	Nrf2 Activator ★ FIC	Friedreich Ataxia (≥16 years)	Oral
Zavzpret	zavegepant	CGRP Receptor Antagonist (Intranasal Gepant)	Acute Migraine Treatment	Intranasal
Daybue	trofinetide	Synthetic Gly-Pro-Glu Tripeptide ★ FIC	Rett Syndrome (≥2 years)	Oral
Zynyz	retifanlimab-dlwr	Anti-PD-1 mAb	Merkel Cell Carcinoma · dMMR Endometrial Cancer	IV
Rezzayo	rezafungin	Echinocandin (Long-acting) ★ FIC	Candidemia and Invasive Candidiasis	IV
Joenja	leniolisib	PI3Kδ Inhibitor	Activated PI3K Delta Syndrome (APDS)	Oral
Qalsody	tofersen	Antisense Oligonucleotide (Anti-SOD1)	ALS with SOD1 Mutation	Intrathecal
Elfabrio	pegunigalsidase alfa-iwxj	PEGylated Recombinant α-Galactosidase A (ERT)	Fabry Disease	IV
Miebo	perfluorohexyloctane	Semifluorinated Alkane (Evaporative Tear Supplement) ★ FIC	Dry Eye Disease (evaporative)	Ophthalmic drops
Epkinly	epcoritamab-bysp	CD3×CD20 Bispecific mAb	Relapsed/Refractory DLBCL (≥3rd line)	SC
Xacduro	sulbactam-durlobactam	β-Lactam/BLI Combination ★ FIC	Acinetobacter baumannii-calcoaceticus pneumonia	IV
Paxlovid	nirmatrelvir / ritonavir	SARS-CoV-2 3CL Protease Inhibitor + CYP3A4 Booster ★ FIC	Mild-Moderate COVID-19 (high-risk adults)	Oral
Posluma	flotufolastat F18	PSMA-Targeted PET Radiopharmaceutical	Prostate Cancer — PSMA PET Imaging	IV
Inpefa	sotagliflozin	Dual SGLT1/SGLT2 Inhibitor ★ FIC	Heart Failure (reduced and preserved EF)	Oral
Columvi	glofitamab-gxbm	CD3×CD20 Bispecific mAb (2:1)	Relapsed/Refractory DLBCL (≥3rd line)	IV
Litfulo	ritlecitinib-jhlz	Dual JAK3/TEC Inhibitor	Severe Alopecia Areata (≥12 years)	Oral
Rystiggo	rozanolixizumab-noli	Anti-FcRn mAb (IgG Recycling Inhibitor)	Generalized Myasthenia Gravis (anti-AChR or anti-MuSK+)	SC
Beyfortus	nirsevimab-alip	Anti-RSV F Protein mAb (Extended Half-life)	RSV Prophylaxis — Neonates and Infants (seasonal)	IM
Vanflyta	quizartinib	FLT3 Inhibitor (Type II)	FLT3-ITD+ AML (1st-line, with chemotherapy)	Oral
Xdemvy	lotilaner ophthalmic	GABA-Cl Channel Antagonist (Topical)	Demodex Blepharitis	Ophthalmic drops
Izervay	avacincaptad pegol	C5 Aptamer (Complement Inhibitor)	Geographic Atrophy Secondary to AMD	Intravitreal
Talvey	talquetamab-tgvs	GPRC5D×CD3 Bispecific mAb ★ FIC	Relapsed/Refractory Multiple Myeloma	SC
Elrexfio	elranatamab-bcmm	BCMA×CD3 Bispecific mAb	Relapsed/Refractory Multiple Myeloma	SC
Sohonos	palovarotene	RARγ Agonist	Fibrodysplasia Ossificans Progressiva (FOP)	Oral
Veopoz	pozelimab-bbfg	Anti-C5 mAb	CD55-Deficient Protein-Losing Enteropathy (CHAPLE disease)	IV/SC
Exxua	gepirone	5-HT1A Partial Agonist (Azapirone)	Major Depressive Disorder	Oral
Pombiliti	cipaglucosidase alfa-atga + miglustat	ERT + Pharmacological Chaperone ★ FIC	Pompe Disease (late-onset, ERT-experienced)	IV
Rivfloza	nedosiran	LDHA-Targeting siRNA	Primary Hyperoxaluria Type 1 (PH1)	SC
Velsipity	etrasimod	S1P Receptor Modulator (S1P1/4/5)	Moderate-to-Severe Ulcerative Colitis	Oral
Zilbrysq	zilucoplan	C5 Inhibitor Peptide (Cyclic Macrocycle)	Generalized Myasthenia Gravis (anti-AChR+)	SC daily
Bimzelx	bimekizumab-bkzx	Dual IL-17A/F Inhibitor ★ FIC	Moderate-to-Severe Plaque Psoriasis	SC
Agamree	vamorolone	Dissociative Glucocorticoid (Modified Steroid)	Duchenne Muscular Dystrophy (4–<18 years)	Oral
Omvoh	mirikizumab-mrkz	Anti-IL-23p19 mAb	Moderate-to-Severe Ulcerative Colitis	IV induction / SC maintenance
Loqtorzi	toripalimab-tpzi	Anti-PD-1 mAb	Recurrent/Metastatic Nasopharyngeal Carcinoma (1L)	IV
Fruzaqla	fruquintinib	VEGFR1/2/3 TKI	Metastatic Colorectal Cancer (≥3rd line)	Oral
Defencath	taurolidine / heparin	Antimicrobial Catheter Lock Solution	Bloodstream Infections in Hemodialysis Catheter Patients	Intraluminal (lock)
Augtyro	repotrectinib	ROS1/TRK Inhibitor (Next-gen)	ROS1+ NSCLC (TKI-naive and -experienced)	Oral
Ryzneuta	eflapegrastim-xnst	Long-acting G-CSF (PEGylated)	Chemotherapy-Induced Neutropenia Prophylaxis	SC
Truqap	capivasertib	AKT Kinase Inhibitor	HR+/HER2- Breast Cancer (AKT pathway-altered, with fulvestrant)	Oral
Ogsiveo	nirogacestat	γ-Secretase Inhibitor	Progressing Desmoid Tumors (adults)	Oral
Fabhalta	iptacopan	Complement Factor B Inhibitor (Oral)	PNH — hemolysis control (monotherapy)	Oral
Filsuvez	birch triterpenes (Oleogel-S10)	Birch Bark Extract (Wound Healing)	Dystrophic & Junctional Epidermolysis Bullosa	Topical
Wainua	eplontersen	GalNAc-Conjugated Anti-TTR ASO	Hereditary Transthyretin Amyloidosis (polyneuropathy)	SC monthly

Leqembi

lecanemab-irmb

Anti-Aβ Protofibril mAb

Indication

Early Alzheimer's Disease — MCI to mild dementia stage with confirmed amyloid pathology

Mechanism

Humanised IgG1 mAb that preferentially binds soluble Aβ protofibrils — the aggregated, pre-plaque oligomeric form most neurotoxic to synapses. Distinct from donanemab, which targets insoluble fibrillar plaques. Fc-receptor-mediated microglial phagocytosis clears amyloid. CLARITY AD trial: 27% slowing of clinical decline (CDR-SB) vs placebo. Received traditional approval Jul 2023.

Dose & Route

IV infusion · 10 mg/kg every 2 weeks · Jan 2023

Brenzavvy

bexagliflozin

SGLT2 Inhibitor

Indication

Type 2 Diabetes Mellitus — adjunct to diet and exercise

Mechanism

Selective inhibitor of SGLT2 in the S1 segment of the proximal renal tubule. Blocks reabsorption of ~90% of filtered glucose, causing glucosuria (~80 g/day) independent of insulin. The resulting osmotic diuresis and natriuresis lower blood pressure and reduce cardiac preload. Class effect: cardiorenal protection mediated partly through reduced tubuloglomerular feedback and ketone body upregulation.

Dose & Route

Oral · 20 mg once daily · Jan 2023

Jaypirca

pirtobrutinib

Non-covalent BTK Inhibitor ★ First-in-Class

Indication

Relapsed/Refractory Mantle Cell Lymphoma — after ≥2 prior therapies including a covalent BTK inhibitor

Mechanism

First non-covalent (reversible) BTK inhibitor. Unlike ibrutinib and acalabrutinib, which form a covalent bond with Cys-481 of BTK, pirtobrutinib binds reversibly to the BTK active site. This preserves full activity against the C481S mutation — the primary resistance mechanism to covalent BTKi. Maintains high selectivity for BTK with low off-target kinase inhibition, translating to a cleaner safety profile (less atrial fibrillation, fewer bleeding events).

Dose & Route

Oral · 200 mg once daily · Jan 2023

Sunlenca

lenacapavir

HIV-1 Capsid Inhibitor ★ First-in-Class

Indication

HIV-1 Infection — heavily treatment-experienced adults with multidrug resistance

Mechanism

First-in-class capsid inhibitor. The HIV capsid (CA) protein orchestrates multiple essential steps: shields viral DNA from innate immune sensors during nuclear transit, facilitates nuclear pore entry (via direct CA–CPSF6 and CA–NUP153 binding), and scaffolds reverse transcription. Lenacapavir binds the CA hexamer/pentamer interface, disrupting assembly and disassembly kinetics at both ends of the viral lifecycle. Exceptionally long t½ (~10–12 weeks) enables once-every-6-months SC maintenance dosing — the longest dosing interval of any ARV.

Dose & Route

Oral 600 mg on Days 1 & 2 (lead-in) → SC 927 mg every 6 months · Dec 2023

Ojjaara

momelotinib

JAK1/JAK2 + ACVR1 Inhibitor ★ First-in-Class

Indication

Myelofibrosis — intermediate- or high-risk with anaemia (JAK-inhibitor naive or JAK-inhibitor experienced)

Mechanism

Inhibits JAK1 and JAK2 (reducing constitutional symptoms and spleen size) while also inhibiting ACVR1 (ALK2) — an activin receptor that drives hepatic hepcidin synthesis via the BMP6–SMAD signalling axis. Hepcidin sequesters iron in macrophages (reducing erythropoiesis). ACVR1 inhibition lowers hepcidin, liberating iron for erythropoiesis. Unlike ruxolitinib, which worsens anaemia (requiring transfusions), momelotinib's ACVR1 activity improves haemoglobin levels and reduces transfusion dependence.

Dose & Route

Oral · 200 mg once daily · Sep 2023

Zurzuvae

zuranolone

Neuroactive Steroid / GABA-A PAM ★ First-in-Class

Indication

Postpartum Depression (PPD) — adults; also approved for Major Depressive Disorder

Mechanism

First oral neuroactive steroid antidepressant. Positive allosteric modulator (PAM) of both synaptic and extrasynaptic GABA-A receptors, especially those containing δ subunits (extrasynaptic). PPD is partly attributed to abrupt neurosteroid (allopregnanolone) withdrawal after delivery, destabilising GABAergic inhibitory tone. Zuranolone restores GABA-A receptor modulation rapidly — onset within 3 days vs 2–4 weeks for SSRIs. Short 14-day course of treatment; not a daily maintenance drug.

Dose & Route

Oral · 50 mg once daily at night for 14 days · Aug 2023

Aphexda

motixafortide

CXCR4 Antagonist ★ First-in-Class

Indication

Haematopoietic Stem Cell Mobilization — in combination with G-CSF for autologous transplantation

Mechanism

Selective, long-acting cyclic peptide antagonist of CXCR4. The CXCL12/SDF-1–CXCR4 axis anchors haematopoietic stem and progenitor cells (HSPCs) to bone marrow stromal niches via VLA-4/VCAM-1 interactions. Motixafortide disrupts this retention, releasing HSPCs into peripheral blood for apheresis collection. Unlike plerixafor (the older CXCR4 antagonist), motixafortide has longer duration of action, enabling better CD34+ cell yield from a single apheresis session in multiple myeloma patients.

Dose & Route

SC · 1.25 mg/kg · given ~10–14 h before each apheresis · Sep 2023

Ngenla

somatrogon-ghla

Long-acting GH Analog

Indication

Pediatric Growth Hormone Deficiency (GHD) — ages 3 years and older

Mechanism

Recombinant human GH (22 kDa) fused to the C-terminal peptide (CTP) of the β-subunit of human chorionic gonadotropin. The CTP extension reduces receptor-mediated clearance, extending t½ from ~2–3 h (daily somatropin) to ~40 h — enabling once-weekly dosing. GH receptor activation drives IGF-1 production in the liver and peripheral tissues, stimulating linear bone growth via growth plate chondrocyte proliferation. Non-inferior to daily somatropin for annualised height velocity (REAL3 trial).

Dose & Route

SC · 0.66 mg/kg once weekly · May 2023

Nexviazyme

avalglucosidase alfa-ngpt

Recombinant Acid α-Glucosidase (ERT)

Indication

Pompe Disease (Glycogen Storage Disease Type II) — late-onset, ages 1 year and older

Mechanism

Enzyme replacement therapy (ERT). Deficiency of lysosomal acid alpha-glucosidase (GAA) causes glycogen accumulation in lysosomes of muscle and cardiac cells. Avalglucosidase carries higher levels of bis-phosphorylated mannose-6-phosphate (bis-M6P) on N-glycans compared to the older alglucosidase alfa (Myozyme/Lumizyme). Enhanced CI-M6PR receptor binding improves cellular uptake into lysosomes by ~5× — increasing intralysosomal glycogen clearance. COMET trial demonstrated superiority over alglucosidase for motor and respiratory outcomes.

Dose & Route

IV infusion · 40 mg/kg every 2 weeks · May 2023

Veoza

fezolinetant

NK3 Receptor Antagonist ★ First-in-Class

Indication

Menopausal Vasomotor Symptoms (moderate-to-severe hot flashes) — adults

Mechanism

First non-hormonal, first NK3R antagonist for menopausal symptoms. In menopause, oestrogen withdrawal disinhibits KNDy neurons (Kisspeptin/Neurokinin B/Dynorphin) of the hypothalamic arcuate nucleus. NKB signals via NK3R on thermoregulatory neurons in the median preoptic area, triggering the vasodilatory heat-dissipation response (hot flash). Fezolinetant selectively blocks NK3R, dampening NKB-driven thermoregulatory dysregulation without hormonal effects — key for patients in whom HRT is contraindicated (e.g. ER+ breast cancer survivors).

Dose & Route

Oral · 45 mg once daily · May 2023

Eohilia

budesonide oral suspension

Topical GI Glucocorticoid

Indication

Eosinophilic Esophagitis (EoE) — ages 11 years and older

Mechanism

Budesonide is a potent glucocorticoid (GR binding affinity ~195× cortisol) with extensive first-pass hepatic metabolism (~90%), minimising systemic exposure. As a viscous oral suspension (not inhaled), it is swallowed slowly to coat and directly contact inflamed oesophageal mucosa — suppressing Th2-driven eosinophilic infiltration (IL-5/IL-13/eotaxin axis). Reduces peak oesophageal eosinophil count to ≤6 eos/hpf. Provides a dietary-restriction-free pharmacological option for EoE, which otherwise requires elimination diets or endoscopic dilation.

Dose & Route

Oral suspension · 2 mg twice daily for 12 weeks · Dec 2023

Orserdu

elacestrant

Oral SERD★ First-in-Class

Indication

ER+/HER2– Breast Cancer — ESR1-mutated, post-CDK4/6 inhibitor progression

Mechanism

First oral SERD approved. Binds ERα with higher affinity than fulvestrant, promoting proteasomal degradation of the receptor. ESR1 mutations (Y537S, D538G) confer ligand-independent ER activation and fulvestrant resistance; elacestrant retains full degrader activity against these mutants — filling the gap after CDK4/6 inhibitor progression.

Dose & Route

Oral · 345 mg once daily · Approved Jan 2023

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Jesduvroq

daprodustat

HIF Prolyl Hydroxylase Inhibitor

Indication

Anemia of CKD — dialysis-dependent adults

Mechanism

Inhibits prolyl hydroxylase domain (PHD) enzymes that normally hydroxylate HIF-1α/HIF-2α, targeting them for VHL-mediated proteasomal degradation. PHD inhibition stabilizes HIF, which activates endogenous EPO gene expression — providing physiologic anemia correction without the supraphysiologic EPO peaks of injection ESAs.

Dose & Route

Oral · Once daily · Approved Feb 2023

Lamzede

velmanase alfa-tycv

Recombinant Alpha-Mannosidase (ERT)★ First-in-Class

Indication

Alpha-Mannosidosis — non-CNS manifestations

Mechanism

Recombinant human lysosomal alpha-mannosidase. Alpha-mannosidosis results from LAMAN gene mutations causing deficient enzyme activity and accumulation of oligomannose N-glycans in lysosomes of multiple cell types. Enzyme replacement delivers functional alpha-mannosidase to lysosomes via M6P receptor uptake, restoring glycoprotein catabolism and reducing storage burden in peripheral tissues.

Dose & Route

IV infusion · Weekly · Approved Feb 2023

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Filspari

sparsentan

Dual AT1R/ET-A Antagonist★ First-in-Class

Indication

IgA Nephropathy — reduce proteinuria (adults)

Mechanism

First dual angiotensin/endothelin receptor antagonist for kidney disease. Combines AT1R blockade (reduces intraglomerular pressure, like ACEi/ARB) with ETA antagonism (reduces mesangial cell proliferation and glomerular inflammation). Dual blockade reduces proteinuria more than RAS inhibition alone — addressing both hemodynamic and inflammatory contributors to IgAN progression.

Dose & Route

Oral · 400 mg once daily · Approved Feb 2023

Skyclarys

omaveloxolone

Nrf2 Activator★ First-in-Class

Indication

Friedreich Ataxia (≥16 years) — first approved treatment

Mechanism

First drug approved for Friedreich ataxia. Synthetic oleanane triterpenoid that activates Nrf2 by covalently binding and inhibiting its repressor KEAP1. Nrf2 activation upregulates antioxidant response genes (HO-1, NQO1, ferritin) in cerebellar neurons and cardiomyocytes — countering the frataxin-deficiency-driven mitochondrial iron overload and reactive oxygen species that cause Friedreich's progressive neurodegeneration.

Dose & Route

Oral · 150 mg once daily · Approved Feb 2023

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Zavzpret

zavegepant

Intranasal CGRP Receptor Antagonist

Indication

Acute Migraine with or without Aura (adults)

Mechanism

First intranasal gepant. Antagonizes CGRP receptor at trigeminal nerve terminals and cranial vasculature — blocking the vasodilatory and neuroinflammatory CGRP signal responsible for migraine pain. Intranasal delivery bypasses nausea/vomiting-impaired oral absorption at attack onset; no vasoconstriction risk unlike triptans (suitable for cardiovascular co-morbidities).

Dose & Route

Intranasal spray · 10 mg per nostril · Single dose at attack onset · Approved Mar 2023

Daybue

trofinetide

Synthetic Gly-Pro-Glu Tripeptide★ First-in-Class

Indication

Rett Syndrome — first approved treatment (≥2 years)

Mechanism

First drug approved for Rett syndrome. Synthetic analog of glycyl-l-prolyl-l-glutamic acid (GPE), a naturally occurring N-terminal tripeptide of IGF-1 with neuroprotective properties. Modulates synaptic dysfunction in MeCP2-deficient neurons by reducing neuroinflammation, supporting dendritic arbor maintenance, and restoring GABAergic/glutamatergic balance disrupted by loss of MeCP2 function.

Dose & Route

Oral solution · Twice daily with food · Approved Mar 2023

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Zynyz

retifanlimab-dlwr

Anti-PD-1 mAb

Indication

Merkel Cell Carcinoma (metastatic/recurrent) · dMMR Endometrial Cancer

Mechanism

IgG4 anti-PD-1 monoclonal antibody blocking the PD-1/PD-L1 checkpoint, restoring T-cell cytotoxic activity in tumor-infiltrating lymphocytes. Merkel cell carcinoma is highly immunogenic (Merkel cell polyomavirus or UV-driven mutations); PD-1 blockade shows durable responses. Also used in dMMR endometrial cancers with high tumour mutational burden conferring checkpoint sensitivity.

Dose & Route

IV infusion · Every 4 weeks · Approved Mar 2023

Rezzayo

rezafungin

Long-Acting Echinocandin★ First-in-Class

Indication

Candidemia and Invasive Candidiasis (adults)

Mechanism

Echinocandin that non-competitively inhibits (1,3)-β-D-glucan synthase, blocking fungal cell wall synthesis. Rezafungin is structurally modified at the C5-amine of the cyclic hexapeptide to confer exceptional stability and a half-life of ~130 hours — enabling once-weekly IV dosing vs. daily caspofungin/micafungin. Concentrated in lysosomes where Candida resides intracellularly.

Dose & Route

IV infusion · Once weekly · Approved Mar 2023

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Joenja

leniolisib

PI3Kδ Inhibitor

Indication

Activated PI3K Delta Syndrome (APDS) — primary immunodeficiency

Mechanism

Selectively inhibits PI3Kδ — the isoform predominantly expressed in hematopoietic cells. APDS is caused by gain-of-function PIK3CD mutations that constitutively activate PI3Kδ in lymphocytes, impairing B-cell maturation, causing T-cell senescence, and recurrent sinopulmonary infections. Leniolisib reduces PI3Kδ-driven AKT/mTOR hyperactivation, restoring lymphocyte function.

Dose & Route

Oral · Twice daily · Approved Mar 2023

Qalsody

tofersen

Anti-SOD1 Antisense Oligonucleotide

Indication

ALS with SOD1 Gene Mutation

Mechanism

Intrathecal ASO that hybridizes to SOD1 mRNA and recruits RNase H1, degrading the transcript and reducing mutant SOD1 protein production. Gain-of-toxic-function mutations in SOD1 cause ~2% of all ALS; reducing mutant protein levels slows or halts neurodegeneration. Delivered intrathecally to achieve sufficient CSF concentrations in motor neurons of the spinal cord.

Dose & Route

Intrathecal injection · Every 28 days (after loading) · Approved Apr 2023

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Elfabrio

pegunigalsidase alfa-iwxj

PEGylated α-Galactosidase A (ERT)

Indication

Fabry Disease (adults, ERT-experienced patients)

Mechanism

PEGylated plant-cell-derived recombinant α-galactosidase A (α-Gal A) that replaces deficient enzyme activity in Fabry disease — reducing lysosomal globotriaosylceramide (Gb3) accumulation in vascular endothelium, kidney, heart, and nervous system. PEGylation extends half-life and reduces immunogenicity vs. agalsidase alfa/beta, allowing every-2-week dosing with potentially better tissue uptake.

Dose & Route

IV infusion · Every 2 weeks · Approved May 2023

Miebo

perfluorohexyloctane

Semifluorinated Alkane Tear Substitute★ First-in-Class

Indication

Dry Eye Disease — evaporative (Meibomian gland dysfunction)

Mechanism

First approved treatment specifically for evaporative dry eye. Water-free semifluorinated alkane that spreads as a monomolecular lipid-mimicking film over the tear surface. Reduces evaporative water loss by acting as a physical barrier at the air–tear interface — replacing the dysfunctional meibomian lipid layer. No preservatives, no active drug; purely physical mechanism. Different from aqueous-deficiency drops (sodium hyaluronate etc.).

Dose & Route

Ophthalmic drops · 1 drop each eye 4× daily · Approved May 2023

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Epkinly

epcoritamab-bysp

CD3×CD20 Bispecific mAb (SC)

Indication

Relapsed/Refractory DLBCL (≥3rd line, after CAR-T or ineligible)

Mechanism

CD3×CD20 bispecific antibody (IgG1) administered subcutaneously. One arm engages CD20 on B-cell lymphoma cells; the other binds CD3ε on T cells — forming an immunological synapse that drives T-cell-mediated tumor lysis. SC delivery (vs. IV glofitamab/mosunetuzumab) enables outpatient administration. Risk of CRS mitigated by step-up dosing.

Dose & Route

SC injection · Weekly × 3 cycles, then Q2–4W · Approved May 2023

Xacduro

sulbactam-durlobactam

β-Lactam + Novel BLI★ FIC (durlobactam)

Indication

Hospital-Acquired / Ventilator-Associated Bacterial Pneumonia — Acinetobacter baumannii-calcoaceticus complex (ABBC)

Mechanism

Sulbactam is both a weak β-lactam antibacterial and an intrinsic inhibitor of Acinetobacter PBP1 and PBP3. Durlobactam is a diazabicyclooctane (DBO) BLI that protects sulbactam from carbapenemases (OXA-type, MBLs) ubiquitous in drug-resistant ABBC. The combination restores sulbactam's killing activity against pan-drug-resistant Acinetobacter strains where carbapenems and polymyxins have failed.

Dose & Route

IV infusion · Every 6 hours · Approved May 2023

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Paxlovid

nirmatrelvir / ritonavir

3CL Protease Inhibitor + CYP3A4 Booster★ FIC (nirmatrelvir)

Indication

Mild-to-Moderate COVID-19 — high-risk adults within 5 days of symptoms

Mechanism

First oral SARS-CoV-2 antiviral. Nirmatrelvir is a peptidomimetic inhibitor of SARS-CoV-2 3CL protease (Mpro) — an enzyme essential for cleaving viral polyprotein precursors into functional replication machinery. Ritonavir inhibits CYP3A4-mediated nirmatrelvir metabolism, boosting plasma concentrations. Active against all Omicron subvariants (Mpro is highly conserved). EUA → full FDA approval May 2023.

Dose & Route

Oral · Twice daily for 5 days · Full approval May 2023

Posluma

flotufolastat F18

PSMA-Targeted PET Tracer

Indication

Prostate Cancer — PSMA PET imaging (staging, recurrence detection)

Mechanism

18F-labeled PSMA-targeted PET radiotracer. Prostate-specific membrane antigen (PSMA/FOLH1) is a glutamate carboxypeptidase highly overexpressed on prostate cancer cells. Flotufolastat binds PSMA with high affinity, concentrating ¹⁸F in PSMA+ lesions and enabling PET detection of primary and metastatic disease at very low PSA levels — critical for directing salvage therapy after radical prostatectomy.

Dose & Route

IV · Single dose before PET scan · Approved May 2023

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Inpefa

sotagliflozin

Dual SGLT1/SGLT2 Inhibitor★ First-in-Class

Indication

Heart Failure (HFrEF and HFpEF) — reduce CV death and HF hospitalizations

Mechanism

Only approved dual SGLT1/SGLT2 inhibitor. SGLT2 inhibition in the proximal tubule provides glucose-lowering, natriuresis, and cardiorenal benefits (same as dapagliflozin/empagliflozin). Additional SGLT1 inhibition in gut enterocytes attenuates postprandial glucose absorption and reduces intestinal glucose-driven inflammation — potentially amplifying cardiac benefit beyond SGLT2 alone. SOLOIST-WHF trial: benefit even when started during acute hospitalization.

Dose & Route

Oral · Once daily · Approved May 2023

Columvi

glofitamab-gxbm

CD3×CD20 Bispecific mAb (2:1 format)★ FIC (2:1 format)

Indication

Relapsed/Refractory DLBCL (≥3rd line)

Mechanism

2:1 bivalent bispecific antibody (two CD20-binding arms, one CD3-binding arm) — engineered with CrossMAb technology. Bivalent CD20 engagement increases tumor avidity and reduces T-cell dose requirements. Obinutuzumab pre-treatment (cycle 1) clears circulating B cells to prevent CRS by reducing the initial tumor burden before glofitamab is introduced. Fixed 12-cycle treatment — unique among bispecifics.

Dose & Route

IV infusion · 12 cycles (step-up dosing) · Approved Jun 2023

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Litfulo

ritlecitinib-jhlz

Dual JAK3/TEC Inhibitor

Indication

Severe Alopecia Areata (≥12 years)

Mechanism

Covalent irreversible inhibitor of JAK3 and the TEC family kinases (BTK, BMX, ITK, TEC, TXK). JAK3 is exclusively expressed in hematopoietic cells (especially T and NK cells) — inhibiting it blocks JAK3/JAK1-mediated cytokine signalling (IL-2, IL-15, IL-21) that drives CD8+ NKG2D+ T-cell attack on hair follicle immune privilege in alopecia areata. TEC inhibition adds a layer of lymphocyte suppression.

Dose & Route

Oral · 50 mg once daily · Approved Jun 2023

Rystiggo

rozanolixizumab-noli

Anti-FcRn mAb

Indication

Generalized Myasthenia Gravis — anti-AChR or anti-MuSK antibody-positive (adults)

Mechanism

Anti-FcRn (neonatal Fc receptor) monoclonal antibody that blocks IgG recycling — the FcRn mechanism that rescues endocytosed IgG from lysosomal degradation, extending its half-life. Blocking FcRn accelerates IgG (including pathogenic anti-AChR/anti-MuSK) catabolism, dramatically lowering autoantibody levels and improving neuromuscular transmission. SC administration; weekly dosing course.

Dose & Route

SC infusion · Weekly for 6 weeks (repeatable) · Approved Jun 2023

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Beyfortus

nirsevimab-alip

Extended-Half-Life Anti-RSV F mAb

Indication

RSV Prophylaxis — neonates and infants entering their first RSV season

Mechanism

Monoclonal antibody targeting the prefusion conformation of the RSV F (fusion) protein — the most conserved and neutralization-sensitive epitope on RSV. An LS mutation in the Fc region extends half-life to ~70 days via enhanced FcRn binding, allowing a single IM injection to cover an entire RSV season. Unlike palivizumab (5 monthly injections, high-risk only), nirsevimab covers all infants with one dose.

Dose & Route

IM injection · Single dose before RSV season · Approved Jul 2023

Vanflyta

quizartinib

FLT3 TKI (Type II)

Indication

FLT3-ITD+ AML — first-line (with standard induction/consolidation chemotherapy)

Mechanism

Type II FLT3 inhibitor that binds the inactive (DFG-out) kinase conformation, blocking constitutive FLT3-ITD signalling (Ras/MAPK, STAT5, PI3K/AKT). FLT3-ITD mutations occur in ~30% of AML and confer poor prognosis. Type II binding gives quizartinib both higher potency and longer target residence time vs. type I FLT3 inhibitors (midostaurin, gilteritinib) at baseline FLT3-ITD. QuANTUM-First trial: OS benefit in newly diagnosed FLT3-ITD+ AML.

Dose & Route

Oral · 40 mg once daily (with/after chemo) · Approved Jul 2023

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Xdemvy

lotilaner ophthalmic solution

Topical GABA-Cl Channel Antagonist★ First-in-Class

Indication

Demodex Blepharitis — first approved treatment

Mechanism

First FDA-approved treatment targeting Demodex mites. Lotilaner is an isoxazoline that blocks invertebrate-specific GABA-gated chloride channels, causing sustained hyperpolarization and paralytic death of Demodex folliculorum and D. brevis — the mites that colonize eyelash follicles and cause chronic blepharitis. Selective for invertebrate vs. mammalian GABA channels → safe for ocular use.

Dose & Route

Ophthalmic drops · 1 drop each eye twice daily · 6 weeks treatment · Approved Jul 2023

Izervay

avacincaptad pegol

C5 Complement Aptamer

Indication

Geographic Atrophy (GA) Secondary to AMD

Mechanism

PEGylated RNA aptamer that binds complement C5 with high specificity, preventing its cleavage to C5a and C5b — blocking terminal complement activation and MAC (membrane attack complex) formation. Drusen-associated complement hyperactivation in AMD drives retinal pigment epithelium death and geographic atrophy expansion; C5 inhibition at the macula slows this process. Monthly intravitreal injection maintains local inhibition.

Dose & Route

Intravitreal injection · Monthly · Approved Aug 2023

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Talvey

talquetamab-tgvs

GPRC5D×CD3 Bispecific mAb★ First-in-Class

Indication

Relapsed/Refractory Multiple Myeloma (≥4 prior lines including anti-CD38, IMiD, PI)

Mechanism

First GPRC5D-targeting bispecific. GPRC5D is an orphan GPCR selectively overexpressed on myeloma plasma cells (low expression in normal tissues except hair follicles). Talquetamab bridges GPRC5D+ myeloma cells to T cells via CD3, triggering cytolysis. Provides a BCMA-independent target for myeloma patients who relapse after anti-BCMA therapies (teclistamab, CAR-T).

Dose & Route

SC injection · Weekly or biweekly (step-up dosing) · Approved Aug 2023

Elrexfio

elranatamab-bcmm

BCMA×CD3 Bispecific mAb

Indication

Relapsed/Refractory Multiple Myeloma (≥4 prior lines including anti-CD38, IMiD, PI)

Mechanism

SC bispecific T-cell engager targeting BCMA (B-cell maturation antigen/TNFRSF17) on myeloma cells and CD3ε on T cells. BCMA is universally expressed on mature plasma cells and myeloma blasts, making it a selective tumor antigen. Elranatamab forces a cytolytic synapse at BCMA+ tumor cells, achieving deep responses including MRD negativity in heavily pre-treated patients. No pre-treatment cytoreduction required.

Dose & Route

SC injection · Weekly × 2 cycles, then Q2W · Approved Aug 2023

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Sohonos

palovarotene

RARγ Agonist★ First-in-Class

Indication

Fibrodysplasia Ossificans Progressiva (FOP) — reduce HO volume

Mechanism

First drug approved for FOP. FOP is caused by gain-of-function ACVR1 mutations causing hyperactive BMP signalling that drives aberrant endochondral ossification of soft tissues. Palovarotene is a selective retinoid RAR-γ agonist — activating RARγ in chondroprogenitor cells suppresses BMP-induced chondrogenesis via downstream crosstalk, preventing the cartilaginous intermediate that leads to heterotopic bone formation.

Dose & Route

Oral · Chronic daily + flare-up dosing · Approved Aug 2023

Veopoz

pozelimab-bbfg

Anti-C5 Monoclonal Antibody

Indication

CHAPLE Disease (CD55-deficient protein-losing enteropathy)

Mechanism

Anti-C5 mAb that inhibits terminal complement activation. CHAPLE (CHAPLE = CHOoking, ALimentary angiodysplasia, P rotein-Losing Enteropathy) is caused by loss-of-function mutations in CD55 (decay-accelerating factor), which normally prevents complement amplification on host cell surfaces. Uncontrolled complement activation in gut vessels → protein-losing enteropathy, thrombosis. C5 inhibition restores control.

Dose & Route

IV loading, then SC weekly · Approved Aug 2023

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Exxua

gepirone

5-HT1A Partial Agonist (Azapirone)

Indication

Major Depressive Disorder (adults)

Mechanism

Azapirone 5-HT1A partial agonist/autoreceptor desensitizer. Acts as an agonist at somatodendritic 5-HT1A autoreceptors in the raphe nuclei and as a partial agonist at postsynaptic 5-HT1A receptors in limbic areas — increasing serotonergic tone without blocking serotonin reuptake. No significant effects on NE, DA, or muscarinic/histamine receptors → cleaner tolerability profile (no weight gain, sexual dysfunction, or sedation typical of SSRIs/SNRIs).

Dose & Route

Oral ER tablet · Twice daily with food · Approved Sep 2023

Pombiliti

cipaglucosidase alfa-atga

ERT + Pharmacological Chaperone★ First-in-Class

Indication

Late-Onset Pompe Disease (ERT-experienced adults) — with miglustat

Mechanism

First ERT + chaperone combination for Pompe disease. Cipaglucosidase alfa is a high-mannose-6-phosphate recombinant GAA with improved lysosomal targeting. Co-administered with miglustat (an iminosugar pharmacological chaperone that stabilizes the GAA enzyme during transit) — the chaperone prevents premature enzyme degradation in the acidic endosome, delivering more active GAA to lysosomes. Superior muscle glycogen clearance vs. alglucosidase alfa alone.

Dose & Route

IV (cipaglucosidase) + Oral (miglustat, 1 hr before infusion) · Every 2 weeks · Approved Sep 2023

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Rivfloza

nedosiran

LDHA-Targeting siRNA

Indication

Primary Hyperoxaluria Type 1 (PH1) — reduce urinary oxalate

Mechanism

GalNAc-conjugated siRNA targeting lactate dehydrogenase A (LDHA) mRNA in hepatocytes. In PH1, AGXT enzyme deficiency causes glyoxalate overflow → conversion to oxalate via LDH. Silencing LDHA reduces this final step in oxalate biosynthesis, lowering urinary oxalate and preventing calcium oxalate nephrolithiasis and nephrocalcinosis. Monthly SC dosing analogous to lumasiran (HOGA1-targeting).

Dose & Route

SC injection · Monthly · Approved Sep 2023

Velsipity

etrasimod

Selective S1P Receptor Modulator

Indication

Moderate-to-Severe Ulcerative Colitis (adults)

Mechanism

Selective S1P1/4/5 receptor modulator. S1P1 on lymphocytes is required for egress from lymph nodes; etrasimod causes functional antagonism (receptor internalization), trapping CCR9+ gut-homing lymphocytes in lymph nodes and reducing their trafficking to inflamed colonic mucosa. Etrasimod has shorter half-life (~30 h) than fingolimod — faster lymphocyte recovery on cessation, important for surgical/infectious scenarios.

Dose & Route

Oral · 2 mg once daily · Approved Oct 2023

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Zilbrysq

zilucoplan

Cyclic Peptide C5 Inhibitor

Indication

Generalized Myasthenia Gravis — anti-AChR antibody-positive (adults)

Mechanism

Macrocyclic peptide that binds complement C5, preventing its cleavage to C5a/C5b and blocking MAC formation at the neuromuscular junction. In anti-AChR+ MG, IgG–complement activation destroys the postsynaptic NMJ architecture. Unlike IV eculizumab, zilucoplan is self-administered daily SC — providing patient convenience. Mechanism is distinct from anti-FcRn therapies (rozanolixizumab/efgartigimod) which reduce total IgG.

Dose & Route

SC injection · Once daily (self-administered) · Approved Oct 2023

Bimzelx

bimekizumab-bkzx

Dual IL-17A/F Inhibitor★ First-in-Class

Indication

Moderate-to-Severe Plaque Psoriasis (adults)

Mechanism

First dual IL-17A/F inhibitor. IL-17A and IL-17F are homodimers and heterodimers that together drive psoriatic keratinocyte proliferation, neutrophil recruitment, and epidermal barrier disruption. Bimekizumab neutralizes both cytokines from a single binding site — a broader blockade than secukinumab/ixekizumab (IL-17A only). Greater skin clearance rates (PASI 90/100) than anti-IL-17A monotherapies in head-to-head trials.

Dose & Route

SC injection · Q4W × 16 weeks, then Q8W · Approved Oct 2023

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Agamree

vamorolone

Dissociative Glucocorticoid★ First-in-Class

Indication

Duchenne Muscular Dystrophy (4–<18 years)

Mechanism

Modified glucocorticoid (steroidal compound with Δ9,11 modification) that dissociates the two main GR signaling pathways. Retains trans-repression of NF-κB/AP-1 (anti-inflammatory benefit in DMD) while reducing trans-activation of metabolic genes (GR-driven GRE transcription) — the source of GC side effects (growth suppression, weight gain, adrenal suppression, bone loss). Designed to preserve anti-inflammatory benefit of corticosteroids in DMD with reduced adverse effects.

Dose & Route

Oral suspension · Once daily · Approved Oct 2023

Omvoh

mirikizumab-mrkz

Anti-IL-23p19 mAb

Indication

Moderate-to-Severe Ulcerative Colitis (adults)

Mechanism

Selectively binds the p19 subunit of IL-23 (IL-23p19), blocking IL-23 signalling without affecting IL-12. IL-23 drives Th17 differentiation and Th17/Th22 effector cell expansion in colonic mucosa — the dominant cytokine pathway in UC. Selective p19 blockade (vs. anti-p40 approaches like ustekinumab that also inhibit IL-12/Th1) preserves immune surveillance while targeting UC-specific Th17 inflammation.

Dose & Route

IV induction (300 mg Q4W × 12W) then SC maintenance (200 mg Q4W) · Approved Oct 2023

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Loqtorzi

toripalimab-tpzi

Anti-PD-1 mAb

Indication

Recurrent/Metastatic Nasopharyngeal Carcinoma — first-line (with gemcitabine-cisplatin)

Mechanism

Anti-PD-1 IgG4 monoclonal antibody. NPC is highly immunogenic (EBV-associated) with a heavily PD-L1-expressing tumor microenvironment — making it among the most PD-1 checkpoint-sensitive cancers. Toripalimab adds immune activation to gemcitabine-cisplatin cytotoxicity (JUPITER-02 trial). First anti-PD-1 approved for NPC; this EBV-associated tumor is particularly prevalent in Southeast Asia and China.

Dose & Route

IV infusion · Every 3 weeks (with chemo then as monotherapy) · Approved Oct 2023

Fruzaqla

fruquintinib

VEGFR1/2/3 TKI

Indication

Metastatic Colorectal Cancer (≥3rd line, after prior chemotherapy and biologics)

Mechanism

Selective small-molecule inhibitor of VEGFR1, VEGFR2, and VEGFR3 kinases — blocking all three receptors that drive pathological tumor angiogenesis and lymphangiogenesis. Highly selective VEGFR profile (minimal off-target kinase inhibition) reduces the toxicity seen with broader TKIs like regorafenib, while maintaining anti-angiogenic efficacy in refractory mCRC (FRESCO-2 trial).

Dose & Route

Oral · 5 mg once daily for 3 weeks on/1 week off · Approved Nov 2023

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Defencath

taurolidine / heparin

Antimicrobial Catheter Lock Solution★ FIC (taurolidine indication)

Indication

Reduce Catheter-Related Bloodstream Infections (CRBSI) in Hemodialysis Patients

Mechanism

Taurolidine is an antimicrobial agent that hydrolyses to taurine derivatives, which react with bacterial/fungal cell wall components and DNA via methylol groups — preventing biofilm formation in catheter lumens. Combined with heparin (for catheter patency). Instilled and left in the catheter between hemodialysis sessions, dramatically reducing CRBSI risk without promoting systemic antimicrobial resistance.

Dose & Route

Intraluminal catheter lock · Instilled after each dialysis session · Approved Nov 2023

Augtyro

repotrectinib

Next-Generation ROS1/TRK Inhibitor

Indication

ROS1+ NSCLC — TKI-naive and TKI-pre-treated (including resistance mutations)

Mechanism

Compact, macrocyclic kinase inhibitor of ROS1 and NTRK1/2/3 (TRK). Small molecular footprint allows binding to ROS1 kinase carrying solvent-front mutations (G2032R) that resist crizotinib, entrectinib, and lorlatinib. Also CNS-penetrant, addressing brain metastases. Designed to overcome acquired resistance across multiple generations of prior ROS1-directed therapy — covering both TKI-naive and -resistant patients.

Dose & Route

Oral · 160 mg once daily (days 1–14), then 160 mg twice daily · Approved Nov 2023

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Ryzneuta

eflapegrastim-xnst

Long-Acting PEGylated G-CSF

Indication

Chemotherapy-Induced Neutropenia Prophylaxis

Mechanism

Recombinant G-CSF (filgrastim) conjugated to a PEG-linked albumin-binding moiety (HSA-binding domain), extending half-life via albumin association. Like pegfilgrastim, stimulates G-CSFR on neutrophil progenitors and mature neutrophils — accelerating neutrophil recovery and reducing duration of severe neutropenia after myelosuppressive chemotherapy. Single SC dose per chemotherapy cycle (same as pegfilgrastim but distinct structural platform).

Dose & Route

SC injection · Single dose per chemo cycle (next day) · Approved Nov 2023

Truqap

capivasertib

AKT Kinase Inhibitor

Indication

HR+/HER2– Breast Cancer — AKT pathway-altered (PIK3CA/AKT1/PTEN), with fulvestrant

Mechanism

Selective inhibitor of all three AKT isoforms (AKT1/2/3) at the ATP-binding site. PIK3CA mutations, AKT1 E17K, and PTEN loss constitutively activate the PI3K→AKT→mTOR survival pathway, driving CDK4/6 inhibitor resistance in HR+ breast cancer. Capivasertib blocks AKT kinase activity, restoring anti-tumor efficacy of fulvestrant in PI3K/AKT/PTEN-altered tumors (CAPItello-291 trial).

Dose & Route

Oral · 400 mg twice daily (4 days on / 3 days off) · Approved Nov 2023

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Ogsiveo

nirogacestat

γ-Secretase Inhibitor

Indication

Progressing Desmoid Tumors (adults)

Mechanism

Selective γ-secretase inhibitor that blocks proteolytic cleavage of Notch receptors into their transcriptionally active intracellular domains. Desmoid tumors harbor APC/β-catenin mutations that activate Wnt/Notch signalling in fibroblasts; Notch activity promotes tumor cell survival and proliferation. Inhibiting γ-secretase interrupts Notch-1/2/3 activation — a mechanism not addressed by cytotoxic agents (DeFi trial: improved PFS and response vs. placebo).

Dose & Route

Oral · 150 mg twice daily · Approved Nov 2023

Fabhalta

iptacopan

Oral Complement Factor B Inhibitor★ First-in-Class

Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH) — hemolysis control (oral monotherapy)

Mechanism

First oral complement inhibitor for PNH. Blocks factor B, the rate-limiting component of the alternative complement pathway C3 convertase (C3bBb). Inhibiting upstream of C3 prevents both intravascular hemolysis (MAC-mediated, targeted by anti-C5 drugs) AND extravascular hemolysis (C3 opsonization) in PNH RBCs — offering a single oral agent that controls both hemolysis mechanisms previously requiring IV eculizumab + oral danicopan.

Dose & Route

Oral · 200 mg twice daily · Approved Dec 2023

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Filsuvez

birch triterpenes (oleogel-S10)

Birch Bark Extract — Wound Healing★ First-in-Class

Indication

Dystrophic and Junctional Epidermolysis Bullosa — wound healing

Mechanism

First approved treatment for EB wound management. Oleogel-S10 contains birch triterpenes (80% betulin from Betula alba bark) in a sunflower oil/polyethylene glycol base. Betulin and related pentacyclic triterpenes promote keratinocyte proliferation and migration, upregulate collagen synthesis, and have anti-inflammatory properties — accelerating wound closure in EB patients who cannot generate normal structural proteins (collagen VII in DEB; laminin-332 in JEB).

Dose & Route

Topical gel · Applied to EB wounds at dressing changes · Approved Dec 2023

Wainua

eplontersen

GalNAc-Conjugated Anti-TTR ASO

Indication

Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN)

Mechanism

GalNAc-conjugated antisense oligonucleotide that hybridizes to TTR mRNA in hepatocytes, inducing RNase H-mediated degradation and reducing TTR protein production. Monthly SC self-injection replaces inotersen (weekly SC injection, monitoring-intensive) and patisiran (quarterly IV infusion). GalNAc conjugation delivers high hepatocyte concentrations via ASGPR-mediated uptake — reducing required dose and allowing home administration without transfusion center visits.

Dose & Route

SC injection · Once monthly (self-administered) · Approved Dec 2023

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Brand Name	Generic Name	Drug Class	Indication	Route
Quviviq	daridorexant	Dual Orexin Receptor Antagonist	Insomnia	Oral
Cibinqo	abrocitinib	JAK1 Inhibitor	Atopic Dermatitis	Oral
Kimmtrak	tebentafusp-tebn	gp100 ImmTAC T-cell Engager ★ FIC	Uveal Melanoma	IV
Vabysmo	faricimab-svoa	Bispecific VEGF-A + Ang-2 Inhibitor ★ FIC	Wet AMD / DME	Intravitreal
Enjaymo	sutimlimab-jome	Classical Complement C1s Inhibitor ★ FIC	Cold Agglutinin Disease	IV
Pyrukynd	mitapivat	Pyruvate Kinase Activator ★ FIC	Pyruvate Kinase Deficiency	Oral
Vonjo	pacritinib	JAK2 / IRAK1 Inhibitor	Myelofibrosis (thrombocytopenic)	Oral
Ztalmy	ganaxolone	GABA-A PAM (Neurosteroid) ★ FIC	CDKL5 Deficiency Disorder	Oral
Opdualag	nivolumab + relatlimab	PD-1 + LAG-3 Dual Checkpoint ★ FIC	Melanoma	IV
Pluvicto	Lu-177 vipivotide	PSMA Radioligand Therapy ★ FIC	mCRPC	IV
Vivjoa	oteseconazole	Selective Fungal CYP51 Inhibitor ★ FIC	Recurrent VVC	Oral
Camzyos	mavacamten	Cardiac Myosin Inhibitor ★ FIC	Obstructive HCM	Oral
Voquezna	vonoprazan + antibiotics	P-CAB + Dual Antibiotic ★ FIC	H. pylori	Oral
Mounjaro	tirzepatide	Dual GIP + GLP-1 Receptor Agonist ★ FIC	Type 2 Diabetes	SC weekly
Vtama	tapinarof	Aryl Hydrocarbon Receptor Agonist ★ FIC	Plaque Psoriasis	Topical
Amvuttra	vutrisiran	GalNAc-siRNA (TTR) ★ FIC	hATTR Polyneuropathy	SC q3 months
Rolvedon	eflapegrastim	Long-acting G-CSF Analog	Febrile Neutropenia Prevention	SC
Sotyktu	deucravacitinib	TYK2 Inhibitor ★ FIC	Plaque Psoriasis	Oral
Spevigo	spesolimab-sbzo	IL-36 Receptor Antagonist ★ FIC	Generalised Pustular Psoriasis	IV
Xenpozyme	olipudase alfa-rpcp	Recombinant Acid Sphingomyelinase (ERT) ★ FIC	ASMD	IV
Daxxify	daxibotulinumtoxinA-lanm	Long-acting Botulinum Toxin A	Glabellar Lines	IM
Elucirem	gadopiclenol	Macrocyclic GBCA (Half-dose)	MRI Contrast	IV
Omlonti	oomidenepag isopropyl	Selective EP2 Receptor Agonist	Glaucoma / OHT	Ophthalmic
Relyvrio	PB / taurursodiol	ER Stress + Mitochondrial Modulator	ALS	Oral
Lytgobi	futibatinib	Irreversible FGFR1–4 Inhibitor	FGFR2+ Cholangiocarcinoma	Oral
Imjudo	tremelimumab	CTLA-4 Blocking mAb	HCC (single priming dose)	IV
Tecvayli	teclistamab-cqyv	BCMA × CD3 Bispecific TCE ★ FIC	Relapsed/Refractory MM	SC
Elahere	mirvetuximab soravtansine	FR-α-targeted ADC ★ FIC	Ovarian Cancer (FRα+)	IV
Tzield	teplizumab-mzwv	CD3-directed T-cell Modulator ★ FIC	Delay T1D Onset	IV
Rezlidhia	olutasidenib	IDH1 Inhibitor	IDH1-mutant AML	Oral
Krazati	adagrasib	KRAS G12C Inhibitor ★ FIC	NSCLC (KRAS G12C)	Oral
Lunsumio	mosunetuzumab-axgb	CD20 × CD3 Bispecific TCE ★ FIC	Follicular Lymphoma	SC
Xenoview	hyperpolarized ¹²⁹Xe	Inhaled MRI Imaging Agent	Pulmonary Ventilation MRI	Inhaled
Briumvi	ublituximab-xiiy	Glycoengineered CD20 mAb	Relapsing MS	IV
NexoBrid	anacaulase-bcdb	Enzymatic Debridement Agent ★ FIC	Severe Burn Eschar	Topical
Terlivaz	terlipressin	Vasopressin V1 Receptor Agonist ★ FIC	Hepatorenal Syndrome Type 1 (HRS-1)	IV

Quviviq

daridorexant

Dual Orexin Receptor Antagonist

Indication

Insomnia — adults

Mechanism

Competitive antagonist of both OX1R and OX2R (orexin receptors). Orexin A/B (hypocretin) are wake-promoting neuropeptides secreted by lateral hypothalamic neurons. Dual blockade attenuates wake-drive without suppressing REM or SWS architecture — key advantage over benzodiazepines and Z-drugs which globally suppress CNS. Unlike suvorexant, daridorexant has a shorter t½ (~8 h), reducing next-morning sedation. No scheduled substance classification.

Dose & Route

Oral · 25 or 50 mg at bedtime · Jan 2022

Cibinqo

abrocitinib

JAK1 Inhibitor

Indication

Moderate-to-severe Atopic Dermatitis — adults and adolescents ≥12 years

Mechanism

Selective JAK1 inhibitor. JAK1 is the principal signalling kinase downstream of IL-4Rα (IL-4, IL-13), IL-31Rα (IL-31, the primary pruritus cytokine), and TSLP receptors — all central to atopic dermatitis pathophysiology. By blocking JAK1-mediated STAT6 phosphorylation, abrocitinib suppresses Th2 cytokine signalling, reduces skin eosinophilia, and markedly relieves itch (PEAK: 43% reduction in itch NRS at week 2). Oral once-daily convenience vs dupilumab injections.

Dose & Route

Oral · 100 or 200 mg once daily · Jan 2022

Kimmtrak

tebentafusp-tebn

gp100-directed T-Cell Engager (ImmTAC) ★ First-in-Class

Indication

Unresectable or Metastatic Uveal Melanoma — HLA-A*02:01+ adults

Mechanism

First-in-class ImmTAC (immune-mobilising monoclonal TCR against cancer). Bifunctional fusion protein: one end is a soluble high-affinity TCR recognising the gp100_280-288 peptide-HLA-A*02:01 complex on melanoma cells; the other is an anti-CD3 scFv that recruits cytotoxic T cells. Unlike CAR-T or standard bispecific antibodies, tebentafusp targets an intracellular antigen (gp100) presented by MHC class I — a novel modality. First ever randomised survival benefit shown in uveal melanoma (mOS 21.7 vs 16.0 months, IMCgp100-202 trial).

Dose & Route

IV infusion · Step-up then 68 mcg weekly · Jan 2022

Vabysmo

faricimab-svoa

Bispecific VEGF-A + Ang-2 Inhibitor ★ First-in-Class

Indication

Neovascular (Wet) AMD and Diabetic Macular Oedema — adults

Mechanism

First bispecific antibody for retinal neovascular disease. Simultaneously inhibits VEGF-A (driving neovascularisation and vascular permeability) and Ang-2 (angiopoietin-2, which destabilises vessel walls by competing with Ang-1 for Tie-2 receptor binding). Dual blockade achieves greater vascular stabilisation than VEGF-A inhibition alone. The Ang-2 axis contributes to chronic inflammation via macrophage activation. Enables extended dosing intervals (up to every 16 weeks after loading) vs monthly ranibizumab.

Dose & Route

Intravitreal injection · 6 mg monthly ×4, then every 8–16 weeks · Jan 2022

Enjaymo

sutimlimab-jome

Classical Complement C1s Inhibitor ★ First-in-Class

Indication

Cold Agglutinin Disease (CAD) — adults to reduce haemolysis

Mechanism

First C1s inhibitor; first treatment approved specifically for CAD. CAD is caused by IgM cold agglutinins that activate the classical complement pathway at low temperatures (acral circulation), driving C3-mediated extravascular haemolysis of red blood cells. Sutimlimab is a humanised IgG4 mAb that selectively inhibits C1s (the serine protease in the C1 complex), blocking C4 cleavage and halting classical pathway activation without impairing the lectin or alternative pathways. CARDINAL trial: 54% of patients achieved haemoglobin ≥12 g/dL.

Dose & Route

IV infusion · 6.5 g (≤75 kg) or 7.5 g (>75 kg) on Days 0 & 7, then every 2 weeks · Feb 2022

Pyrukynd

mitapivat

Pyruvate Kinase (PKR) Activator ★ First-in-Class

Indication

Pyruvate Kinase Deficiency (PKD) — haemolytic anaemia in adults

Mechanism

First pyruvate kinase activator; first disease-modifying treatment for PKD. PKD is caused by loss-of-function mutations in PKLR (pyruvate kinase liver/red cell isoform), reducing ATP production in red blood cells. RBC energy depletion leads to ion pump failure, membrane rigidity, and haemolysis. Mitapivat allosterically activates mutant PKR, increasing enzyme activity and restoring RBC ATP and 2,3-DPG levels — reducing haemolysis and improving haemoglobin. Active against most PKLR missense mutations.

Dose & Route

Oral · 5 mg twice daily (titrated to 20 mg twice daily) · Feb 2022

Vonjo

pacritinib

JAK2 / IRAK1 Inhibitor

Indication

Myelofibrosis — intermediate- or high-risk with severe thrombocytopenia (platelets <50×10⁹/L)

Mechanism

Dual inhibitor of JAK2 (myeloproliferative signalling, mutant JAK2 V617F) and IRAK1 (interleukin-1 receptor-associated kinase 1, involved in NF-κB-driven inflammatory signalling in myeloid cells). Key differentiator: unlike ruxolitinib, pacritinib does not inhibit JAK1 — sparing thrombopoietin/JAK1/STAT5 signalling, thus preserving megakaryocyte function and allowing use in severely thrombocytopenic patients who cannot tolerate ruxolitinib. PERSIST-2 trial: spleen volume reduction in platelet counts <50×10⁹/L.

Dose & Route

Oral · 200 mg twice daily · Feb 2022

Ztalmy

ganaxolone

GABA-A Receptor Positive Allosteric Modulator ★ First-in-Class

Indication

Seizures associated with CDKL5 Deficiency Disorder (CDD) — ages 2 and older

Mechanism

First treatment approved specifically for CDD. Ganaxolone is a synthetic neurosteroid analogue of allopregnanolone. Acts as a PAM at synaptic and extrasynaptic GABA-A receptors (particularly δ-subunit containing receptors), enhancing chloride ion influx and neuronal inhibition. CDD is caused by mutations in the CDKL5 gene (cyclin-dependent kinase-like 5), which impairs neuronal dendritic morphology and synaptic function — resulting in early-onset, refractory epilepsy. Oral liquid formulation allows paediatric use.

Dose & Route

Oral solution · Three times daily (weight-based dosing) · Mar 2022

Opdualag

nivolumab + relatlimab-rmbw

Dual Checkpoint Inhibitor (PD-1 + LAG-3) ★ First-in-Class

Indication

Unresectable or Metastatic Melanoma — adults and children ≥12 years

Mechanism

First LAG-3–targeting combination; first dual PD-1/LAG-3 checkpoint blockade. Nivolumab (anti-PD-1) + relatlimab (anti-LAG-3) in a fixed-dose co-formulation. LAG-3 (Lymphocyte Activation Gene-3) is an inhibitory receptor expressed on exhausted TILs alongside PD-1; it binds MHC class II and fibrinogen-like protein 1 on tumour cells/APCs. Concurrent blockade of both checkpoints synergistically restores T-cell function — additive to PD-1 alone (RELATIVITY-047 trial: mPFS 10.1 vs 4.6 months).

Dose & Route

IV infusion · Nivolumab 480 mg + relatlimab 160 mg every 4 weeks · Mar 2022

Pluvicto

lutetium Lu 177 vipivotide tetraxetan

PSMA-targeted Radioligand Therapy ★ First-in-Class

Indication

PSMA-positive Metastatic Castration-Resistant Prostate Cancer (mCRPC) — after ARPI and taxane

Mechanism

First PSMA-targeted radioligand therapy (RLT); landmark in theranostics. A small-molecule PSMA ligand (vipivotide/PSMA-617) conjugated to lutetium-177 (β-emitter, range ~2 mm in tissue). PSMA is overexpressed on prostate cancer cells. After IV infusion, the ligand binds PSMA on tumour cells; β-radiation causes double-strand DNA breaks, inducing tumour cell death while sparing adjacent normal tissue. VISION trial: 38% reduction in risk of death vs standard-of-care. Companion diagnostic: ⁶⁸Ga-PSMA-11 PET/CT.

Dose & Route

IV infusion · 7.4 GBq every 6 weeks × 6 cycles · Mar 2022

Vivjoa

oteseconazole

Selective Fungal CYP51 Inhibitor (VCAN) ★ First-in-Class

Indication

Recurrent Vulvovaginal Candidiasis (rVVC) — non-pregnant adult women

Mechanism

First selective fungal CYP51 inhibitor approved for rVVC prevention. Oteseconazole inhibits fungal lanosterol 14α-demethylase (CYP51) — essential for ergosterol synthesis in the Candida cell membrane — with >4,000-fold selectivity over human CYP51. Unlike standard azoles (fluconazole), which also inhibit human CYP51 (steroid synthesis), oteseconazole avoids hormonal side effects and hepatotoxicity. Provides oral prophylaxis to prevent recurrence following an acute episode, addressing a major unmet need in chronic rVVC.

Dose & Route

Oral · Initial treatment course then weekly maintenance dosing · Apr 2022

Camzyos

mavacamten

Cardiac Myosin Inhibitor ★ First-in-Class

Indication

Obstructive Hypertrophic Cardiomyopathy (oHCM) — adults with NYHA class II–III symptoms

Mechanism

First cardiac myosin inhibitor; first mechanism-targeted therapy for HCM. oHCM is driven by excess cross-bridge formation between myosin heavy chains and actin filaments, generating hypercontractility and LVOT obstruction. Mavacamten selectively inhibits β-cardiac myosin ATPase, reducing the proportion of myosin heads in the force-producing ('super-relaxed') state — decreasing actin-myosin cross-bridge formation, contractility, and LVOT gradient. EXPLORER-HCM trial: 27% achieved primary endpoint (LVOT ΔP <30 mmHg + NYHA improvement).

Dose & Route

Oral · 2.5–15 mg once daily (titrated by echocardiographic LVOT gradient and LVEF) · Apr 2022

Voquezna

vonoprazan / amoxicillin / clarithromycin

P-CAB + Dual Antibiotic (H. pylori Triple Therapy) ★ First-in-Class

Indication

H. pylori Infection — triple therapy (14-day course)

Mechanism

First potassium-competitive acid blocker (P-CAB) approved in the US. Vonoprazan reversibly and competitively inhibits the K⁺-binding site of H⁺/K⁺-ATPase on gastric parietal cells, achieving faster and more complete acid suppression than proton pump inhibitors (PPIs) which require acid activation. Stable in acidic pH (unlike PPIs). Maintained acid suppression creates a more favourable microenvironment for clarithromycin/amoxicillin antibacterial activity against H. pylori. Higher eradication rates than PPI-based triple therapy (84% vs 79% in Phase 3).

Dose & Route

Oral · Vonoprazan 20 mg + amoxicillin 1000 mg + clarithromycin 500 mg twice daily × 14 days · May 2022

Mounjaro

tirzepatide

Dual GIP + GLP-1 Receptor Agonist ★ First-in-Class

Indication

Type 2 Diabetes Mellitus — adjunct to diet and exercise

Mechanism

First dual GIP/GLP-1 receptor agonist (twincretin). A single synthetic peptide that simultaneously agonises both GIP receptor (GIPR) and GLP-1 receptor (GLP-1R) — the two major incretin receptors. GLP-1R agonism: reduces glucagon, slows gastric emptying, enhances glucose-dependent insulin secretion, suppresses appetite. GIPR agonism: potentiates insulin secretion synergistically with GLP-1R, improves insulin sensitivity in adipose tissue, and modulates energy balance via hypothalamic GIPR signalling. SURPASS trials: HbA1c reduction up to 2.5%, weight loss up to 11.9 kg at 40 weeks. Later approved for obesity (Zepbound, 2023).

Dose & Route

SC · 2.5 mg once weekly (titrated up to 15 mg) · May 2022

Vtama

tapinarof

Aryl Hydrocarbon Receptor (AhR) Agonist ★ First-in-Class

Indication

Plaque Psoriasis — adults

Mechanism

First topical AhR agonist for dermatology. Tapinarof activates the aryl hydrocarbon receptor (AhR), a ligand-activated transcription factor. In keratinocytes, AhR activation suppresses Th17/Th22-driven pro-inflammatory cytokines (IL-17, IL-22, TNF-α), upregulates skin barrier proteins (filaggrin, loricrin), and reduces oxidative stress via Nrf2 pathway induction. A steroid-free topical mechanism that can be applied to sensitive skin areas (face, intertriginous zones). Phase 3 trials: 36–40% IGA 0/1 response.

Dose & Route

Topical cream 1% · Once daily · May 2022

Amvuttra

vutrisiran

siRNA — Transthyretin (TTR) synthesis silencer ★ First-in-Class

Indication

Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN) — adults

Mechanism

First quarterly siRNA; improved successor to patisiran. Uses GalNAc-siRNA conjugate technology (N-acetylgalactosamine targeting hepatic asialoglycoprotein receptors) for direct liver delivery — eliminating the need for LNP formulation and pre-medication. Once internalised, the siRNA is loaded into RISC (RNA-induced silencing complex), cleaving TTR mRNA — reducing hepatic production of both mutant and wild-type transthyretin by ~80–90%. Less mutant/WT TTR in circulation means less amyloid deposition in peripheral nerves. HELIOS-A trial: superior to placebo on neuropathy progression.

Dose & Route

SC · 25 mg once every 3 months · Jun 2022

Rolvedon

eflapegrastim

Long-acting G-CSF Analog

Indication

Chemotherapy-induced Febrile Neutropenia Prevention — adults

Mechanism

Eflapegrastim is a novel G-CSF analog consisting of recombinant G-CSF conjugated to an Fc fragment via a polyethylene glycol (PEG) linker. The Fc domain extends circulation half-life by FcRn-mediated recycling, allowing once-per-chemotherapy-cycle dosing (like pegfilgrastim). G-CSF receptor (G-CSFR) stimulation drives proliferation, differentiation, and activation of neutrophil progenitors in bone marrow. Non-inferior to pegfilgrastim for neutrophil recovery (ADVANCE trial).

Dose & Route

SC · 13.2 mg once per chemotherapy cycle (24 h after chemo) · Sep 2022

Sotyktu

deucravacitinib

TYK2 Inhibitor ★ First-in-Class

Indication

Moderate-to-Severe Plaque Psoriasis — adults

Mechanism

First-in-class TYK2 inhibitor; first non-JAK1/2/3 kinase inhibitor for psoriasis. Selectively allosterically inhibits TYK2 (tyrosine kinase 2) by binding its pseudokinase (JH2) regulatory domain — a unique allosteric mechanism that avoids the ATP-binding site occupied by JAK1/2/3 inhibitors. TYK2 mediates downstream signalling of IL-23 (→STAT3→Th17 differentiation) and IL-12 (→STAT4→Th1). Blockade of TYK2 suppresses IL-17A/F and IFN-γ production — the key drivers of psoriatic plaques. High selectivity over JAK1/2/3: no black-box warning for thrombosis/malignancy/mortality.

Dose & Route

Oral · 6 mg once daily · Sep 2022

Spevigo

spesolimab-sbzo

IL-36 Receptor Antagonist ★ First-in-Class

Indication

Generalised Pustular Psoriasis (GPP) Flares — adults

Mechanism

First IL-36R antagonist; first approved treatment specifically for GPP flares. GPP is a rare, potentially life-threatening neutrophilic skin disorder driven by IL-36 cytokine hyperactivation (IL-36α/β/γ signalling via IL-36R). IL-36 activates NF-κB and MAPK pathways in keratinocytes, amplifying IL-8, TNF, and CXCL1 release — recruiting neutrophils and generating sterile pustules. Mutations in IL36RN (IL-36RA) are the most common genetic driver. Spesolimab blocks IL-36R, halting the inflammatory cascade. Effisayil-1 trial: 54% clearance vs 6% placebo at week 1.

Dose & Route

IV infusion · 900 mg single dose for acute flare; optional 300 mg SC at week 1 · Sep 2022

Xenpozyme

olipudase alfa-rpcp

Recombinant Acid Sphingomyelinase (ERT) ★ First-in-Class

Indication

Acid Sphingomyelinase Deficiency (ASMD) — non-CNS manifestations in adults and children

Mechanism

First enzyme replacement therapy for ASMD (Niemann-Pick disease type A/B). ASMD is caused by mutations in SMPD1 (sphingomyelin phosphodiesterase 1), leading to sphingomyelin accumulation in lysosomes of hepatic, splenic, and pulmonary macrophages. Olipudase alfa is recombinant human ASM (rhASM) that is taken up via M6P receptors into lysosomes, hydrolysing sphingomyelin to ceramide + phosphocholine. Reduces hepatosplenomegaly and improves pulmonary function and lipid profiles. Dose-escalation protocol required to avoid inflammatory reactions from rapid sphingomyelin clearance.

Dose & Route

IV infusion · Escalating doses every 2 weeks (target 3 mg/kg adults / 1–3 mg/kg paediatric) · Aug 2022

Daxxify

daxibotulinumtoxinA-lanm

Long-acting Botulinum Toxin Type A

Indication

Moderate-to-Severe Glabellar Lines — adults

Mechanism

A formulation of botulinum toxin type A stabilised with a peptide excipient (RTP004) that replaces human serum albumin and extends duration of action. Cleaves SNAP-25 (synaptosomal-associated protein 25), preventing vesicular ACh release at the neuromuscular junction → temporary flaccid paralysis of procerus and corrugator supercilii muscles. Extended duration vs onabotulinumtoxinA (onabot): median 24 weeks (vs ~12 weeks) in Phase 3 — enabling less frequent treatment cycles.

Dose & Route

IM injection · 40 units total (split across 5 injection sites) every ~6 months · Sep 2022

Elucirem

gadopiclenol

Macrocyclic Gadolinium-Based Contrast Agent (GBCA)

Indication

CNS MRI Contrast Enhancement — adults and children ≥2 years

Mechanism

Macrocyclic gadolinium chelate with high kinetic stability (transmetalation resistance) and relaxivity (~3× that of conventional linear GBCAs per mmol Gd). Higher relaxivity allows effective imaging at half the gadolinium dose (0.05 mmol/kg vs 0.1 mmol/kg for standard agents), reducing total gadolinium deposition. Gadolinium shortens T1 relaxation time of surrounding water protons, increasing signal intensity in areas of blood-brain barrier disruption (e.g. tumours, inflammation, demyelination).

Dose & Route

IV · 0.05 mmol/kg (half-dose protocol) · Sep 2022

Omlonti

oomidenepag isopropyl

Selective EP2 Prostanoid Receptor Agonist

Indication

Open-Angle Glaucoma or Ocular Hypertension — adults

Mechanism

Selective agonist of the prostanoid EP2 receptor (prostaglandin E2 receptor subtype 2). Unlike latanoprost/bimatoprost which act on FP receptors increasing uveoscleral outflow, EP2 agonism acts through a distinct pathway: increases trabecular meshwork outflow (conventional pathway) as well as uveoscleral outflow via cAMP/adenylyl cyclase signalling. Causes less iris pigmentation and conjunctival hyperaemia than FP agonists. First EP2-selective ophthalmic agent approved.

Dose & Route

Ophthalmic solution 0.002% · One drop once daily in the evening · Sep 2022

Relyvrio

sodium phenylbutyrate / taurursodiol

Mitochondrial ER Stress Modulator Combo

Indication

ALS (Amyotrophic Lateral Sclerosis) — adults

Mechanism

Fixed-dose combination of two agents: sodium phenylbutyrate (PB) — a chemical chaperone that reduces endoplasmic reticulum (ER) stress by enhancing protein folding and suppressing the unfolded protein response (UPR); and taurursodiol (TUDCA) — tauroursodeoxycholic acid, a bile acid that inhibits mitochondrial apoptosis by preventing cytochrome c release and activating Akt/mTOR survival pathways. Motor neuron death in ALS involves both ER stress and mitochondrial dysfunction. CENTAUR trial: 2.32 ALSFRS-R point benefit at 24 weeks. Withdrawn from US market 2024 after PHOENIX trial failed to replicate benefit.

Dose & Route

Oral · 3 g PB + 1 g taurursodiol once daily then twice daily · Sep 2022

Lytgobi

futibatinib

Irreversible FGFR1–4 Inhibitor

Indication

FGFR2 Fusion/Rearrangement-positive Intrahepatic Cholangiocarcinoma — previously treated adults

Mechanism

Covalently and irreversibly binds Cys492 in the FGFR kinase domain, inhibiting all four FGFR isoforms (FGFR1-4). Unlike reversible FGFR inhibitors (pemigatinib), covalent binding overcomes resistance mutations at the kinase gatekeeper residue (V565L). FGFR2 gene fusions/rearrangements occur in 15–20% of intrahepatic cholangiocarcinomas and constitutively activate downstream RAS-MAPK and PI3K-AKT signalling driving tumour growth. FOENIX-CCA2 trial: ORR 41.7% in FGFR2 fusion-positive iCCA.

Dose & Route

Oral · 20 mg once daily (3 weeks on / 1 week off) · Sep 2022

Imjudo

tremelimumab

CTLA-4 Blocking mAb

Indication

Unresectable Hepatocellular Carcinoma (uHCC) — in combination with durvalumab (1 priming dose)

Mechanism

Anti-CTLA-4 IgG2 mAb. CTLA-4 is a co-inhibitory receptor on T cells that competes with CD28 for B7-1/B7-2 ligands on APCs, dampening early T-cell activation in lymph nodes. Tremelimumab blockade augments priming and expansion of tumour-reactive T cells. Given as a single 300 mg priming dose combined with durvalumab (anti-PD-L1) in the HIMALAYA regimen — a 'T+D STRIDE' protocol. Single high-dose tremelimumab achieves CTLA-4 blockade early; durvalumab maintains peripheral and tumoral T-cell activity long-term. OS 16.4 vs 13.8 months (HIMALAYA trial).

Dose & Route

IV · 300 mg single priming dose (with durvalumab 1500 mg), then durvalumab alone every 4 weeks · Oct 2022

Tecvayli

teclistamab-cqyv

BCMA × CD3 Bispecific T-Cell Engager ★ First-in-Class

Indication

Relapsed or Refractory Multiple Myeloma — after ≥4 prior lines of therapy

Mechanism

First BCMA-directed bispecific T-cell engager (TCE) approved. Simultaneously binds BCMA (B-cell maturation antigen) on myeloma plasma cells and CD3ε on T cells, forming an immunological synapse that redirects T-cell cytotoxicity to myeloma cells — independent of TCR specificity. Different from anti-BCMA ADCs (belantamab): recruits endogenous T cells rather than delivering cytotoxic payload. MajesTEC-1 trial: ORR 63% in heavily pretreated patients. Cytokine release syndrome (CRS) is the main toxicity; step-up dosing protocol mitigates risk.

Dose & Route

SC · Step-up: 0.06 mg/kg then 0.3 mg/kg, then 1.5 mg/kg weekly · Oct 2022

Elahere

mirvetuximab soravtansine-gynx

FR-α-targeted Antibody-Drug Conjugate ★ First-in-Class

Indication

Folate Receptor-α (FRα)-positive Platinum-Resistant Ovarian Cancer — adults (prior bevacizumab)

Mechanism

First FRα-directed ADC approved. Anti-FRα mAb (mirvetuximab) conjugated via a cleavable sulfo-SPDB linker to DM4 (maytansinoid microtubule inhibitor). FRα is overexpressed in epithelial ovarian cancers, providing tumour-selective targeting. After internalisation via FRα-mediated endocytosis, lysosomal cleavage releases DM4, which binds β-tubulin (inhibiting polymerisation) → mitotic arrest → apoptosis. Bystander effect: released DM4 diffuses to adjacent FRα-negative cells. SORAYA trial: ORR 32.4% in platinum-resistant disease.

Dose & Route

IV infusion · 6 mg/kg (adjusted ideal body weight) every 3 weeks · Nov 2022

Tzield

teplizumab-mzwv

CD3-directed mAb (T-cell Modulator) ★ First-in-Class

Indication

Delay Onset of Stage 3 Type 1 Diabetes — adults and children ≥8 years at high risk (stage 2 T1D)

Mechanism

First drug to delay T1D onset; first immunotherapy to modify T1D natural history. Anti-CD3ε mAb that modulates T-cell function without depleting T cells. Mechanism: (1) partially blocks TCR signalling in auto-reactive effector T cells targeting beta cells; (2) expands a population of exhausted regulatory T cells (TIGIT+ KLRG1+ Teffs) that suppress islet autoimmunity. The 14-day IV course induces a 'regulatory reset'. TN-10 trial: median 2-year delay in clinical T1D onset among stage 2 high-risk individuals (islet autoantibody+ plus dysglycaemia).

Dose & Route

IV infusion · 14-day course; escalating doses from 51–1030 mcg/m²/day · Nov 2022

Rezlidhia

olutasidenib

IDH1 Inhibitor

Indication

IDH1-mutated Relapsed/Refractory AML — adults

Mechanism

Small-molecule inhibitor of mutant IDH1 (isocitrate dehydrogenase 1). IDH1 gain-of-function mutations (R132H/C/S) produce the oncometabolite 2-hydroxyglutarate (2-HG), which inhibits TET2 demethylases and Jumonji-domain histone demethylases → hypermethylation of DNA/histones → differentiation block ('epigenetic lock') on myeloid progenitors. Olutasidenib competitively inhibits mutant IDH1, reducing 2-HG and restoring differentiation. Complete remission (CR + CRh) rate: 35% in the registrational trial. Second approved IDH1 inhibitor (after ivosidenib).

Dose & Route

Oral · 150 mg twice daily · Dec 2022

Krazati

adagrasib

KRAS G12C Inhibitor ★ First-in-Class

Indication

KRAS G12C-mutated Non-Small Cell Lung Cancer (NSCLC) — previously treated adults

Mechanism

Second KRAS G12C inhibitor approved (after sotorasib), with broader activity. Covalently and irreversibly binds Cys12 of the KRAS G12C mutant in the inactive GDP-bound state, locking KRAS in an off-state and preventing GEF-mediated GDP→GTP exchange. KRAS G12C occurs in ~13% of NSCLC. Additional activity against CNS metastases (CNS ORR 33.3% in KRYSTAL-12 vs sotorasib's limited CNS penetration). Also shows activity against KRAS G12C in CRC (in combination with cetuximab).

Dose & Route

Oral · 600 mg twice daily · Dec 2022

Lunsumio

mosunetuzumab-axgb

CD20 × CD3 Bispecific T-Cell Engager ★ First-in-Class

Indication

Relapsed or Refractory Follicular Lymphoma — after ≥2 prior lines of therapy

Mechanism

First subcutaneously administered CD20×CD3 bispecific antibody; first bispecific approved for FL. IgG1 bispecific mAb that crosslinks CD20 on B-cell lymphoma with CD3ε on T cells, forming a cytotoxic synapse. Redirects polyclonal T cells to kill CD20+ tumour cells without requiring prior T-cell priming or genetic engineering (unlike CAR-T). Fixed-duration 8-cycle regimen. GO29781 trial: ORR 80% in R/R FL with 60% CR. Manageable CRS profile with step-up dosing in cycle 1.

Dose & Route

SC · Step-up dosing in cycle 1 (1/2/60 mg on days 1/8/15), then 30 mg on day 1 of cycles 2–8 · Dec 2022

Xenoview

hyperpolarized xenon Xe-129

Inhaled Pulmonary Imaging Agent

Indication

Evaluation of Pulmonary Ventilation — adults

Mechanism

Xenon-129 gas is hyperpolarised via spin-exchange optical pumping (SEOP), increasing the magnetic resonance signal by up to 100,000-fold compared to thermally polarised ¹²⁹Xe. When inhaled, hyperpolarised ¹²⁹Xe distributes through ventilated airways and alveoli; ventilation defects (from emphysema, asthma, IPF, PE) appear as signal voids on MRI. Provides functional ventilation mapping without ionising radiation (unlike V/Q scintigraphy). ¹²⁹Xe also dissolves into lung tissue and red blood cells, enabling gas exchange imaging.

Dose & Route

Inhaled · 0.5–1.0 L dose immediately before breath-hold MRI acquisition · Dec 2022

Briumvi

ublituximab-xiiy

CD20-directed mAb (Glycoengineered)

Indication

Relapsing Forms of Multiple Sclerosis (RMS) — adults

Mechanism

Chimeric IgG1 anti-CD20 mAb with glycoengineered Fc region (reduced fucosylation) for enhanced ADCC (antibody-dependent cellular cytotoxicity) potency. Targets the same CD20 epitope as ofatumumab/ocrelizumab but binds a distinct, non-overlapping B-cell epitope region on CD20. Depletes mature B cells (CD20+) via ADCC and CDC (complement-dependent cytotoxicity), reducing CNS and peripheral B-cell-driven neuroinflammation. Short infusion time (1 hour after first dose) — faster than ocrelizumab (3.5 h). ULTIMATE I&II trials: 59% reduction in ARR vs teriflunomide.

Dose & Route

IV infusion · 150 mg on Days 1 & 15, then 450 mg every 24 weeks · Dec 2022

NexoBrid

anacaulase-bcdb

Enzymatic Eschar Debridement Agent ★ First-in-Class

Indication

Eschar Removal in Severe Burns — adults (thermal burns ≥ TBSA 4% deep partial/full thickness)

Mechanism

First enzymatic debridement agent for burns approved in the US. A concentrate of proteolytic enzymes (bromelain-derived, enriched for stem bromelain from pineapple) that selectively degrades denatured collagen and fibrin in burn eschar while sparing viable underlying tissue. Applied under an occlusive dressing for 4 hours; enzymatic action removes eschar without sharp surgical debridement — preserving viable dermis, reducing blood loss, avoiding the need for multiple surgical procedures, and potentially improving wound healing outcomes. DETECT trial: successful debridement in 89% vs 4.7% vehicle.

Dose & Route

Topical · 2 g/100 cm² applied to wound under occlusion × 4 hours (single application per target wound) · Dec 2022

Terlivaz

terlipressin

Vasopressin V1 Receptor Agonist★ First-in-Class

Indication

Hepatorenal Syndrome Type 1 (HRS-1) — improve kidney function in adults

Mechanism

First drug approved specifically for HRS-1 in the US. Prodrug cleaved in vivo to lysine vasopressin, a potent V1 receptor agonist on splanchnic arteriolar smooth muscle. In decompensated cirrhosis, severe splanchnic vasodilation reduces effective circulating volume, triggering renal vasoconstriction and HRS. Terlipressin induces splanchnic vasoconstriction — reversing the hemodynamic mechanism that drives renal failure — improving GFR and serum creatinine. Long used in Europe; FDA approval Sept 2022.

Dose & Route

IV bolus or continuous infusion · Approved Sep 2022

🔍

Brand Name	Generic Name	Drug Class	Indication	Route
Adbry	tralokinumab-ldrm	Anti-IL-13 mAb	Atopic Dermatitis (moderate-severe)	SC
Leqvio	inclisiran	GalNAc-siRNA (PCSK9) ★ FIC	HeFH / ASCVD	SC q6 months
Vyvgart	efgartigimod alfa-fcab	FcRn Inhibitor ★ FIC	Generalised MG	IV
Tezspire	tezepelumab-ekko	Anti-TSLP mAb ★ FIC	Severe Asthma	SC q4 weeks
Cytalux	pafolacianine	Folate Receptor Fluorescent Imaging ★ FIC	Ovarian Cancer Surgery	IV
Livtencity	maribavir	CMV UL97 Kinase Inhibitor ★ FIC	Post-transplant CMV	Oral
Voxzogo	vosoritide	CNP Analog (NPR2/GC-B agonist) ★ FIC	Achondroplasia	SC daily
Besremi	ropeginterferon alfa-2b	Mono-PEGylated IFN-α2b	Polycythemia Vera	SC
Scemblix	asciminib	STAMP Inhibitor (Myristoyl Pocket) ★ FIC	Ph+ CML (BTKi-resistant / T315I)	Oral
Tavneos	avacopan	C5aR1 (Complement C5a Receptor) Inhibitor ★ FIC	ANCA Vasculitis	Oral
Livmarli	maralixibat	Ileal Bile Acid Transporter (IBAT) Inhibitor ★ FIC	Alagille Syndrome Pruritus	Oral
Qulipta	atogepant	Oral CGRP Receptor Antagonist ★ FIC	Episodic Migraine Prevention	Oral
Xywav	Ca/Mg/K/Na oxybates	GABA-B Agonist / CNS Depressant	Idiopathic Hypersomnia	Oral
Rezurock	belumosudil	ROCK2 Inhibitor ★ FIC	Chronic GvHD	Oral
Exkivity	mobocertinib	EGFR Exon 20ins TKI ★ FIC	NSCLC (EGFR ex20ins)	Oral
Vanflyta	quizartinib	FLT3 Inhibitor (type II)	FLT3-ITD+ AML	Oral
Amondys 45	casimersen	PMO ASO (Exon 45 Skipper)	DMD (exon 45 amenable)	IV
Saphnelo	anifrolumab-fnia	Type I IFN Receptor Blocker ★ FIC	Systemic Lupus Erythematosus	IV
Tivdak	tisotumab vedotin-tftv	Tissue Factor-Directed ADC	Recurrent/Metastatic Cervical Cancer	IV
Skytrofa	lonapegsomatropin-tcgd	Once-Weekly Long-Acting GH	Pediatric Growth Hormone Deficiency	SC weekly
Korsuva	difelikefalin	Peripheral κ-Opioid Receptor Agonist ★ FIC	Pruritus in CKD (hemodialysis)	IV (post-dialysis)
Welireg	belzutifan	HIF-2α Inhibitor ★ FIC	VHL Disease — RCC, CNS hemangioblastomas, pNET	Oral
Bylvay	odevixibat	IBAT Inhibitor ★ FIC	Pruritus in PFIC (Progressive Familial Intrahepatic Cholestasis)	Oral
Fexinidazole	fexinidazole	Nitroimidazole Antiparasitic ★ FIC	African Trypanosomiasis (Human African Sleeping Sickness)	Oral
Kerendia	finerenone	Nonsteroidal Mineralocorticoid Receptor Antagonist ★ FIC	CKD in Type 2 Diabetes — reduce CV and renal events	Oral
Rylaze	asparaginase erwinia chrysanthemi (recombinant)-rywn	Recombinant Asparaginase	ALL/LBL — hypersensitive to E. coli-derived asparaginase	IM/IV
Aduhelm	aducanumab-avwa	Anti-Aβ Amyloid Plaque-Directed mAb	Alzheimer Disease (early)	IV
Brexafemme	ibrexafungerp	Triterpenoid Glucan Synthase Inhibitor ★ FIC	Vulvovaginal Candidiasis	Oral
Lybalvi	olanzapine / samidorphan	D2 Antagonist + Peripheral Opioid Antagonist ★ FIC	Schizophrenia · Bipolar I Disorder	Oral
Truseltiq	infigratinib	FGFR1/2/3 Kinase Inhibitor	FGFR2-Fusion Cholangiocarcinoma (2nd line)	Oral
Lumakras	sotorasib	KRAS G12C Covalent Inhibitor ★ FIC	KRAS G12C-Mutated NSCLC (2nd line)	Oral
Pylarify	piflufolastat F18	PSMA-Targeted PET Tracer	Prostate Cancer — PSMA PET Imaging	IV
Rybrevant	amivantamab-vmjw	EGFR×MET Bispecific mAb ★ FIC	NSCLC — EGFR Exon 20 Insertion Mutations	IV
Empaveli	pegcetacoplan	C3 Inhibitor (Cyclic Peptide) ★ FIC	Paroxysmal Nocturnal Hemoglobinuria (PNH)	SC
Zynlonta	loncastuximab tesirine-lpyl	CD19 ADC (PBD-Dimer Payload)	Relapsed/Refractory DLBCL (≥2 prior lines)	IV
Jemperli	dostarlimab-gxly	Anti-PD-1 mAb	dMMR Endometrial Cancer · MSI-H Solid Tumors	IV
Nextstellis	drospirenone / estetrol	Progestin + Estetrol (Native Estrogen) ★ FIC	Oral Contraception	Oral
Qelbree	viloxazine ER	Selective NE Reuptake Inhibitor / 5-HT Modulator ★ FIC	ADHD (6–17 years)	Oral
Zegalogue	dasiglucagon	Glucagon Receptor Agonist (Aqueous Formulation)	Severe Hypoglycemia (diabetes, ≥6 years)	SC auto-injector
Ponvory	ponesimod	Selective S1P1 Receptor Modulator	Relapsing Multiple Sclerosis	Oral
Fotivda	tivozanib	VEGFR1/2/3 TKI (4th line)	Relapsed/Refractory RCC (≥2 prior lines)	Oral
Azstarys	serdexmethylphenidate / dexmethylphenidate	Prodrug CNS Stimulant	ADHD (≥6 years)	Oral
Nulibry	fosdenopterin	Cyclic Pyranopterin Monophosphate (cPMP) Substrate Replacement ★ FIC	Molybdenum Cofactor Deficiency Type A (MOCD-A)	IV
Cosela	trilaciclib	CDK4/6 Inhibitor (Myeloprotective) ★ FIC	Myelosuppression Prophylaxis in Extensive SCLC Chemotherapy	IV
Evkeeza	evinacumab-dgnb	Anti-ANGPTL3 mAb ★ FIC	Homozygous Familial Hypercholesterolemia (HoFH)	IV
Tepmetko	tepotinib	MET Exon 14 TKI	METex14-Mutated NSCLC	Oral
Lupkynis	voclosporin	Calcineurin Inhibitor (Enhanced CNI)	Active Lupus Nephritis	Oral
Cabenuva	cabotegravir / rilpivirine LA	INSTI + NNRTI (Long-Acting Injectable) ★ FIC	HIV-1 (virologically stable, switch to monthly IM)	IM monthly
Verquvo	vericiguat	Soluble Guanylate Cyclase Stimulator	Heart Failure with Reduced EF (post-decompensation)	Oral
Pepaxto	melphalan flufenamide	Peptide-Drug Conjugate Alkylating Agent	Relapsed/Refractory Multiple Myeloma (heavily pre-treated)	IV
Zynlonta	loncastuximab tesirine-lpyl	CD19 ADC (PBD Dimer)	Relapsed/Refractory DLBCL	IV

Adbry

tralokinumab-ldrm

Anti-IL-13 Monoclonal Antibody

Indication

Moderate-to-Severe Atopic Dermatitis — adults

Mechanism

Fully human IgG4 mAb that specifically neutralises IL-13 (not IL-4). IL-13 is the dominant driver of skin barrier disruption (downregulating FLG, LOR, claudins), Th2 skewing, and IgE class switching in AD. Unlike dupilumab (which blocks IL-4Rα shared by both IL-4 and IL-13), tralokinumab acts downstream, selectively blocking IL-13 signalling (via IL-13Rα1/IL-4Rα complex) without affecting IL-4-only pathways. This selective approach may spare some beneficial IL-4 functions while targeting the primary itch-and-barrier driver in chronic AD. ECZTRA trials: 16–22% IGA 0/1 at week 16.

Dose & Route

SC · 600 mg loading, then 300 mg every 2 weeks · Dec 2021

Leqvio

inclisiran

siRNA — PCSK9 Synthesis Silencer ★ First-in-Class

Indication

Heterozygous Familial Hypercholesterolaemia or ASCVD — adjunct to maximally tolerated statin

Mechanism

First siRNA for hypercholesterolaemia; first twice-yearly lipid-lowering injection. Uses GalNAc conjugate for hepatocyte-selective delivery. Loaded into RISC in hepatocyte cytoplasm → cleaves PCSK9 mRNA → blocks synthesis (not secreted protein). Reduced hepatocyte PCSK9 means more LDL receptors recycle to the surface → increased LDL-C clearance. Unique: once RISC is loaded, the silencing effect persists for ~6 months, enabling SC dosing on Day 1, Month 3, then every 6 months. LDL-C lowering ~50%. Differentiated from evolocumab/alirocumab (anti-PCSK9 mAbs dosed more frequently).

Dose & Route

SC · Day 1, Month 3, then every 6 months · Dec 2021

Vyvgart

efgartigimod alfa-fcab

FcRn Inhibitor ★ First-in-Class

Indication

Generalised Myasthenia Gravis (gMG) — AChR antibody-positive adults

Mechanism

First FcRn inhibitor approved; novel mechanism for antibody-driven disease. Fragment of human IgG1 Fc region (Fc fragment) engineered with enhanced binding affinity for the neonatal Fc receptor (FcRn). FcRn normally salvages IgG from lysosomal degradation by recycling it to the cell surface. Efgartigimod competitively occupies FcRn, diverting endogenous IgG (including pathogenic anti-AChR antibodies) to lysosomal degradation → reduces total IgG and disease-driving autoantibodies. Cyclic 4-weekly infusions (not continuous) with repeat cycles as needed. ADAPT trial: 67.7% responded in AChR+ cohort.

Dose & Route

IV infusion · 10 mg/kg weekly × 4 doses/cycle; repeat cycles as needed · Dec 2021

Tezspire

tezepelumab-ekko

Anti-TSLP Monoclonal Antibody ★ First-in-Class

Indication

Severe Asthma — adults and adolescents ≥12 years (add-on maintenance)

Mechanism

First anti-TSLP biologic; broadest applicability across all severe asthma phenotypes. TSLP (Thymic Stromal Lymphopoietin) is an epithelial 'master switch' cytokine released in response to allergens, viruses, pollutants, and non-allergic triggers. It activates dendritic cells, ILC2s, mast cells, and Th2 cells, initiating the full type-2 (and type-3) inflammatory cascade — upstream of IL-4, IL-5, IL-13, and IgE. Unlike anti-IL-5/IL-4 biologics, tezepelumab works even in low-eosinophil, non-allergic asthma. NAVIGATOR trial: 70% reduction in exacerbations regardless of baseline eosinophil count.

Dose & Route

SC · 210 mg every 4 weeks · Dec 2021

Cytalux

pafolacianine

Folate Receptor-targeted Fluorescent Imaging Agent ★ First-in-Class

Indication

Intraoperative Identification of Ovarian Cancer Lesions — adults (adjunct to standard surgical technique)

Mechanism

First folate receptor-targeted optical imaging agent. Pafolacianine is folate conjugated to a near-infrared (NIR) fluorescent dye (S0456). After IV infusion, it binds folate receptor α (FRα) — highly expressed on ovarian cancer cells but minimally on normal tissue. Accumulates in FRα+ tumour deposits and is visualised under NIR light during surgery, helping surgeons identify lesions not visible to the naked eye. Improves complete cytoreduction: 26.9% of patients had at least one additional lesion found vs standard white-light surgery. Enables targeted excision of occult metastases.

Dose & Route

IV infusion · 0.025 mg/kg single dose 1–9 h before procedure · Nov 2021

Livtencity

maribavir

CMV UL97 Protein Kinase Inhibitor ★ First-in-Class

Indication

Post-Transplant Cytomegalovirus (CMV) Infection/Disease — refractory or resistant to ganciclovir, valganciclovir, foscarnet, or cidofovir

Mechanism

First-in-class CMV UL97 kinase inhibitor with a distinct mechanism. Inhibits the CMV-encoded protein kinase UL97, which phosphorylates viral proteins involved in DNA replication and nuclear egress of virions. Critically: UL97 does not need to phosphorylate the drug for activity (unlike ganciclovir, which requires UL97 phosphorylation for activation). Unique dual effect: also inhibits human CDK7 and RNA polymerase II C-terminal domain phosphorylation, but the primary antiviral effect is UL97 inhibition. Active against ganciclovir-resistant strains with UL54 or UL97 mutations (C592G, A594V). SOLSTICE trial: 55.7% clearance vs 23.9% IAT.

Dose & Route

Oral · 400 mg twice daily × 8 weeks · Nov 2021

Voxzogo

vosoritide

C-type Natriuretic Peptide (CNP) Analog ★ First-in-Class

Indication

Achondroplasia — children ≥2 years with open growth plates

Mechanism

First pharmacological treatment targeting the FGFR3 pathway in achondroplasia. Vosoritide is a biologically stable analog of CNP (C-type natriuretic peptide). CNP activates the natriuretic peptide receptor 2 (NPR2/GC-B) on growth plate chondrocytes, elevating cGMP → activating cGMP-dependent protein kinase (PKG) → phosphorylating and inhibiting RAF1 → suppressing the FGFR3→MEK→ERK signalling cascade that drives growth plate chondrocyte differentiation block. Achondroplasia is caused by gain-of-function FGFR3 mutations (predominantly G380R). Annualised height velocity increase: 1.57 cm/year vs placebo.

Dose & Route

SC · 15 mcg/kg once daily · Nov 2021

Besremi

ropeginterferon alfa-2b-njft

Mono-PEGylated Interferon Alfa-2b

Indication

Polycythemia Vera (PV) — adults

Mechanism

Mono-PEGylated (single PEG attachment point) recombinant IFN-α2b with improved tolerability and extended half-life vs non-pegylated IFN-α. Binds type I IFN receptor (IFNAR1/2) → JAK1/TYK2 → STAT1/STAT2 → ISG expression. Mechanism in PV: (1) direct antiproliferative effect on JAK2 V617F-mutant myeloid progenitors; (2) preferentially suppresses clonal expansion of JAK2 V617F+ haematopoietic stem cells over normal HSCs (allele burden reduction); (3) immune-mediated clearance of mutant clones. PROUD-PV/CONTINUATION-PV trial: superior molecular response (allele burden reduction) vs hydroxyurea.

Dose & Route

SC · 100 mcg every 2 weeks (initial dose, titrated to response) · Nov 2021

Scemblix

asciminib

STAMP Inhibitor of BCR-ABL1 ★ First-in-Class

Indication

Philadelphia Chromosome-positive CML — previously treated with ≥2 TKIs; or T315I-mutant CML

Mechanism

First-in-class STAMP inhibitor (Specifically Targeting the ABL Myristoyl Pocket). Binds the ABL1 myristoyl pocket (allosteric site) rather than the ATP-binding site used by all prior BCR-ABL TKIs (imatinib, dasatinib, etc.). This unique binding site is: (1) away from all known ATP-site resistance mutations; (2) allosterically locks ABL1 in an inactive conformation. High potency against T315I (the gatekeeper mutation resistant to all first- and second-generation TKIs) at 200 mg twice daily. Combination potential with ATP-site TKIs (different binding sites). ASCEMBL trial: MCyR at 24 weeks 25.5% vs 13.2% for bosutinib.

Dose & Route

Oral · 40 mg twice daily (standard) or 200 mg twice daily (T315I mutation) · Oct 2021

Tavneos

avacopan

Complement C5a Receptor (C5aR1) Inhibitor ★ First-in-Class

Indication

ANCA-associated Vasculitis (EGPA or MPA) — adults (in combination with standard therapy)

Mechanism

First oral complement inhibitor for vasculitis; first C5aR1 antagonist approved. ANCA-associated vasculitis (AAV) involves complement C5a-driven priming of neutrophils: ANCA IgG activates primed neutrophils, releasing proteases, ROS, and NETs that damage small vessel walls. Avacopan selectively blocks C5aR1 (complement receptor 5a receptor 1/CD88) on neutrophils and macrophages, preventing C5a-mediated degranulation without blocking the full terminal complement cascade (C5b-9 intact for host defence). Enables glucocorticoid tapering/elimination. ADVOCATE trial: non-inferiority then superiority to high-dose prednisolone for remission at 52 weeks.

Dose & Route

Oral · 30 mg twice daily · Oct 2021

Livmarli

maralixibat

Ileal Bile Acid Transporter (IBAT) Inhibitor ★ First-in-Class

Indication

Cholestatic Pruritus in Alagille Syndrome — children ≥1 year

Mechanism

First IBAT inhibitor approved; first systemic treatment for Alagille syndrome pruritus. Alagille syndrome (ALGS, JAG1 mutations) causes paucity of intrahepatic bile ducts → cholestasis → bile acid accumulation → intractable pruritus. Maralixibat inhibits the apical sodium-dependent bile acid transporter (ASBT/IBAT) in the terminal ileum, blocking entero-hepatic recycling of bile acids → increased faecal bile acid excretion → reduced serum bile acid levels → reduced pruritus. ICONIC trial: 70% reduction in pruritus vs baseline. Also in development for PFIC.

Dose & Route

Oral solution · 380 mcg/kg once daily (weight-based) · Sep 2021

Qulipta

atogepant

Oral CGRP Receptor Antagonist (Gepant) ★ First-in-Class

Indication

Episodic Migraine Prevention — adults

Mechanism

First oral CGRP receptor antagonist approved specifically for migraine prevention. CGRP (calcitonin gene-related peptide) is released from trigeminal nerve terminals during migraine and acts on CGRP receptors on meningeal blood vessels and neurons, causing vasodilation and central sensitisation. Atogepant blocks CLR/RAMP1 (the CGRP receptor complex), preventing CGRP-mediated vasodilation and nociceptive signalling. Unlike rimegepant/ubrogepant (acute treatment), atogepant is dosed daily for prevention. Unlike anti-CGRP mAbs (erenumab etc.), atogepant is small-molecule oral with no injection. ADVANCE trial: 56–61% ≥50% responder rate.

Dose & Route

Oral · 10, 30, or 60 mg once daily · Sep 2021

Xywav

Ca/Mg/K/Na oxybates

CNS Depressant / GABA-B Agonist

Indication

Idiopathic Hypersomnia (IH) and Narcolepsy — adults

Mechanism

Mixed-cation salt formulation of γ-hydroxybutyrate (GHB/oxybate): calcium, magnesium, potassium, and sodium salts combined to reduce sodium content by 92% vs Xyrem (sodium oxybate) — clinically important for patients with hypertension or sodium-restricted diets. GHB/oxybate acts as an endogenous CNS depressant: binds GABA-B receptors (primary therapeutic effect) and GHB receptors (weak affinity). In IH: consolidates nocturnal sleep → reduces excessive daytime sleepiness, prolonged sleep inertia, and naps. First drug approved specifically for IH (pivotal trial: 75% reduced EDS, 47% IHss score improvement).

Dose & Route

Oral solution · Bedtime dose + second dose 2.5–4 h later · Aug 2021

Rezurock

belumosudil

ROCK2 Inhibitor ★ First-in-Class

Indication

Chronic Graft-versus-Host Disease (cGVHD) — after ≥2 prior lines of therapy

Mechanism

First-in-class ROCK2 (Rho-associated coiled-coil kinase 2) inhibitor. ROCK2 regulates T-cell and B-cell inflammatory signalling in cGVHD: (1) promotes Th17/Tfh differentiation and IL-21/IL-17 production (pathogenic in fibrotic cGVHD); (2) inhibits Treg differentiation. Belumosudil preferentially inhibits ROCK2 over ROCK1, suppressing STAT3 phosphorylation (IL-17/IL-21 axis), reducing germinal centre B-cell activity, and restoring Treg:Th17 balance. Also reduces downstream TGF-β-mediated fibrosis signalling. ROCKstar trial: ORR 74% (200 mg once daily) in steroid-refractory cGVHD.

Dose & Route

Oral · 200 mg once daily · Jul 2021

Exkivity

mobocertinib

EGFR Exon 20 Insertion–selective TKI ★ First-in-Class

Indication

EGFR Exon 20 Insertion-mutated NSCLC — previously treated adults

Mechanism

First TKI specifically designed and approved for EGFR exon 20 insertion mutations. EGFR exon 20 insertions (ex20ins) occur in ~2–3% of NSCLC; they insert 3–21 bp in-frame near the C-helix of EGFR, creating a structural constraint that paradoxically reduces binding affinity for first/second/third-generation EGFR TKIs while keeping the kinase constitutively active. Mobocertinib is an irreversible TKI designed to fit the altered binding pocket of ex20ins-EGFR (particularly insertions near A767, S768, V769). Covalently modifies Cys797 of EGFR. ORR 28% in post-platinum ex20ins NSCLC. Note: subsequently superseded by amivantamab+chemo (PAPILLON trial); accelerated approval withdrawn 2023.

Dose & Route

Oral · 160 mg once daily · Sep 2021

Vanflyta

quizartinib

FLT3 Inhibitor (type II)

Indication

FLT3-ITD-positive AML — newly diagnosed adults (induction + consolidation + maintenance)

Mechanism

Highly selective, potent inhibitor of FLT3 (FMS-like tyrosine kinase 3) with type II binding (targets inactive DFG-out conformation). FLT3-ITD (internal tandem duplication) mutations occur in 25% of AML and activate FLT3 constitutively, driving STAT5/PI3K/RAS-MAPK-mediated proliferation and blocked myeloid differentiation. Quizartinib is ~1,000× more selective for FLT3 vs other kinases. Second-generation FLT3 inhibitor with improved potency over midostaurin (first FLT3i). QuANTUM-First trial: OS benefit when added to induction/consolidation + maintenance (OS 31.9 vs 15.1 months in FLT3-ITD AML).

Dose & Route

Oral · 35.4 mg once daily (during chemo and maintenance) · Jul 2021

Amondys 45

casimersen

Antisense Oligonucleotide (Exon 45 Skipper)

Indication

Duchenne Muscular Dystrophy (DMD) — amenable to exon 45 skipping (~8% of DMD patients)

Mechanism

Phosphorodiamidate morpholino oligomer (PMO) that hybridises to exon 45 splice donor site of dystrophin pre-mRNA, causing it to be skipped during pre-mRNA splicing. In patients with frameshift mutations where exon 45 skipping restores the reading frame, this produces a shorter but partially functional dystrophin protein (as in Becker MD). Casimersen is the first exon 45 skipping drug; exon 45 is one of the most common exon deletion hotspots in DMD (~8% of patients). ESSENCE trial: increased dystrophin protein levels on muscle biopsy (1.74% vs 0.92% of normal).

Dose & Route

IV infusion · 30 mg/kg once weekly · Feb 2021

Saphnelo

anifrolumab-fnia

Type I Interferon Receptor (IFNAR1) Blocking mAb ★ First-in-Class

Indication

Moderate-to-Severe Systemic Lupus Erythematosus (SLE) — adults on standard therapy

Mechanism

First type I IFN receptor blocker approved for SLE. Fully human IgG1κ mAb targeting subunit 1 of the type I interferon receptor (IFNAR1), blocking all type I IFN subtypes (IFN-α, IFN-β, IFN-ω). SLE is characterised by a prominent 'interferon signature' — elevated type I IFN drives plasmacytoid DC activation, auto-reactive B-cell maturation, and loss of self-tolerance. TULIP-2 trial: 47.8% BICLA response (vs 31.5% placebo) in patients with high baseline IFN signature. Unlike belimumab (anti-BLyS), anifrolumab acts upstream at the IFN receptor, blocking the master driver of SLE autoimmunity.

Dose & Route

IV infusion · 300 mg every 4 weeks · Aug 2021

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Tivdak

tisotumab vedotin-tftv

Tissue Factor ADC

Indication

Recurrent/Metastatic Cervical Cancer — previously treated

Mechanism

Antibody-drug conjugate targeting Tissue Factor (TF/CD142), a coagulation initiator protein overexpressed on cervical cancer cells. The anti-TF antibody delivers monomethyl auristatin E (MMAE, a microtubule-disrupting payload) selectively to TF+ tumor cells via internalization → MMAE release → mitotic arrest and apoptosis. TF is highly expressed in ~80% of cervical cancers, making it a selective ADC target in this indication.

Dose & Route

IV infusion · Every 3 weeks · Approved Sep 2021

Welireg

belzutifan

HIF-2α Inhibitor★ First-in-Class

Indication

VHL Disease — RCC, CNS hemangioblastomas, pancreatic neuroendocrine tumors

Mechanism

First HIF-2α inhibitor approved. VHL mutations cause inability to degrade HIF-2α → constitutive HIF-2α/ARNT transcriptional activity driving angiogenesis (VEGF), erythropoiesis (EPO), and tumor growth. Belzutifan directly inhibits HIF-2α heterodimerization with ARNT — blocking HIF-2α transcriptional output. Oral alternative to surgery for VHL-associated tumors that would otherwise require multiple repeated resections.

Dose & Route

Oral · 120 mg once daily · Approved Aug 2021

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Kerendia

finerenone

Nonsteroidal MRA★ First-in-Class

Indication

CKD Associated with Type 2 Diabetes — reduce CV and renal progression events

Mechanism

First nonsteroidal mineralocorticoid receptor antagonist approved for CKD. Steroidal MRAs (spironolactone, eplerenone) cause hyperkalemia and sex-hormone side effects. Finerenone's nonsteroidal structure binds MR with higher selectivity and different co-regulator recruitment — producing stronger anti-inflammatory/antifibrotic effects in kidney/heart with less hyperkalemia. FIDELIO-DKD/FIGARO-DKD: reduced composite renal and CV endpoints in T2D-CKD on top of standard RAS blockade.

Dose & Route

Oral · 10–20 mg once daily (guided by eGFR/K+) · Approved Jul 2021

Empaveli

pegcetacoplan

C3 Complement Inhibitor★ First-in-Class

Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH) — first C3 inhibitor

Mechanism

First complement C3 inhibitor approved. PEGylated cyclic peptide that binds C3 and C3b, blocking the entire complement cascade at C3 — preventing both intravascular hemolysis (MAC, targeted by eculizumab) and extravascular hemolysis (C3b-opsonized RBC clearance by macrophages). Twice-weekly SC self-injection; superior to eculizumab in hemoglobin maintenance in PEGASUS trial by also controlling extravascular component.

Dose & Route

SC infusion (via pump) · Twice weekly · Approved May 2021

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Lumakras

sotorasib

KRAS G12C Covalent Inhibitor★ First-in-Class

Indication

KRAS G12C-Mutated NSCLC — previously treated (first approved KRAS inhibitor)

Mechanism

First KRAS inhibitor approved in any cancer. KRAS was historically "undruggable" — no deep binding pockets in wild-type KRAS. The G12C mutation creates a unique cysteine in the switch-II pocket accessible only in the GDP-bound inactive state. Sotorasib covalently alkylates Cys12, locking KRAS G12C in the inactive GDP-bound state and blocking RAS/MAPK downstream signalling. Proof-of-concept for targeting oncogenic KRAS.

Dose & Route

Oral · 960 mg once daily · Approved May 2021

Rybrevant

amivantamab-vmjw

EGFR×MET Bispecific mAb★ First-in-Class

Indication

NSCLC with EGFR Exon 20 Insertion Mutations — post-platinum chemotherapy

Mechanism

Bispecific IgG1 antibody targeting both EGFR and MET extracellular domains. Blocks EGFR/MET ligand-dependent signalling, induces receptor downregulation, and — via its Fc region — recruits immune effectors (NK cells, macrophages) for ADCC/ADCP. EGFR exon 20 insertions create a structural alteration resistant to approved EGFR TKIs; amivantamab's mechanism bypasses this by blocking the receptor extracellularly rather than competing at the ATP-binding site.

Dose & Route

IV infusion · Weekly × 4, then Q2W · Approved May 2021

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Jemperli

dostarlimab-gxly

Anti-PD-1 mAb

Indication

dMMR Recurrent/Advanced Endometrial Cancer · MSI-H Solid Tumors

Mechanism

Anti-PD-1 IgG4 monoclonal antibody restoring T-cell anti-tumor immunity. dMMR (mismatch repair-deficient) endometrial cancers harbor a high mutational burden creating numerous neoantigens, making them exquisitely sensitive to PD-1 checkpoint blockade. Dostarlimab achieved 100% complete response rate in small Phase 2 dMMR locally advanced rectal cancer trial (2022 NEJM) — garnering broad attention beyond endometrial cancer.

Dose & Route

IV infusion · Every 3 weeks × 4 doses, then Q6W · Approved Apr 2021

Zynlonta

loncastuximab tesirine-lpyl

CD19 ADC (PBD-Dimer Payload)

Indication

Relapsed/Refractory Diffuse Large B-Cell Lymphoma (≥2 prior lines)

Mechanism

Anti-CD19 antibody conjugated to SG3199, a pyrrolobenzodiazepine (PBD) dimer that cross-links DNA strands at the N2-guanine position — creating interstrand crosslinks that arrest DNA replication and transcription with extreme potency (picomolar activity). CD19 is universally expressed on B-cell lymphomas; PBD-dimers are among the most potent ADC payloads, effective even at low ADC expression.

Dose & Route

IV infusion · Every 3 weeks · Approved Apr 2021

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Cosela

trilaciclib

Transient Myeloprotective CDK4/6 Inhibitor★ First-in-Class

Indication

Chemotherapy-Induced Myelosuppression Prophylaxis — extensive-stage SCLC

Mechanism

First myeloprotective CDK4/6 inhibitor — administered BEFORE chemotherapy. Transiently arrests hematopoietic stem and progenitor cells (HSPCs) in G1 phase by inhibiting CDK4/6 → Rb hypophosphorylation → cell cycle pause. HSPCs in G1 are insensitive to S/G2-targeting chemotherapy, protecting the bone marrow reservoir. After chemo clearance, trilaciclib washout allows HSPC re-entry into cycle — restoring hematopoiesis faster and reducing neutropenia/anemia/thrombocytopenia duration.

Dose & Route

IV infusion · 4 hours BEFORE chemotherapy on each chemo day · Approved Feb 2021

Evkeeza

evinacumab-dgnb

Anti-ANGPTL3 mAb★ First-in-Class

Indication

Homozygous Familial Hypercholesterolemia (HoFH) — add-on to other lipid-lowering therapy

Mechanism

First anti-ANGPTL3 therapy approved. Blocks angiopoietin-like protein 3 (ANGPTL3), an endogenous inhibitor of lipoprotein lipase (LPL) and hepatic lipase. Evinacumab inhibits ANGPTL3 → releases LPL → dramatically increases TG and LDL-C hydrolysis/clearance. Crucially, works independently of LDL receptors — making it the only effective LDL-lowering agent in HoFH patients with no functional LDL receptors (unresponsive to statins, ezetimibe, PCSK9 inhibitors).

Dose & Route

IV infusion · 15 mg/kg every 4 weeks · Approved Feb 2021

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Cabenuva

cabotegravir / rilpivirine LA

INSTI + NNRTI (Long-Acting IM)★ First-in-Class

Indication

HIV-1 — virologically suppressed adults (switch from daily oral regimen)

Mechanism

First complete long-acting injectable HIV regimen. Cabotegravir (integrase strand transfer inhibitor) + rilpivirine (NNRTI) co-formulated as nanosuspensions for IM gluteal injection. Both drugs form intramuscular depots with sustained release over weeks, achieving therapeutic concentrations with monthly (or bimonthly) injections. Eliminates daily pill burden — transformative for adherence and HIV stigma management. ATLAS/FLAIR/ATLAS-2M trials: non-inferior to daily oral regimens.

Dose & Route

IM injection · Monthly (or every 2 months with higher doses) · Approved Jan 2021

Verquvo

vericiguat

Soluble Guanylate Cyclase Stimulator

Indication

Heart Failure with Reduced EF — following recent worsening/hospitalization

Mechanism

Stimulates soluble guanylate cyclase (sGC) independent of NO — directly activates sGC and sensitizes it to available NO. Increased intracellular cGMP in vascular smooth muscle and cardiomyocytes causes vasodilation and anti-fibrotic/anti-hypertrophic effects in the failing heart. Mechanistically distinct from sacubitril/valsartan, SGLT2i, or beta-blockers — adding a complementary neurohumoral pathway for post-decompensation HFrEF risk reduction (VICTORIA trial).

Dose & Route

Oral · 2.5–10 mg once daily with food · Approved Jan 2021

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Lupkynis

voclosporin

Enhanced Calcineurin Inhibitor

Indication

Active Lupus Nephritis (Class III/IV ± V) — with mycophenolate and corticosteroids

Mechanism

Modified calcineurin inhibitor (cyclosporin analog with a single amino-acid modification) that inhibits calcineurin → blocks NFAT dephosphorylation → reduces IL-2-driven T-cell activation. Additional direct stabilizing effect on podocyte actin cytoskeleton (via synaptopodin pathway) — protecting glomerular filtration architecture independently of its immunosuppressive activity. Linear PK (unlike cyclosporin) allows dosing without therapeutic drug monitoring.

Dose & Route

Oral · 23.7 mg twice daily · Approved Jan 2021

Tepmetko

tepotinib

MET Exon 14 Kinase Inhibitor

Indication

Metastatic NSCLC with MET Exon 14 Skipping Mutations

Mechanism

Highly selective MET kinase inhibitor. MET exon 14 skipping mutations delete the juxtamembrane domain containing Cbl-binding sites responsible for MET ubiquitination and degradation → receptor accumulation → constitutive MET kinase signalling (RAS/MAPK, PI3K/AKT, STAT3). Tepotinib blocks MET kinase activity, shutting down downstream oncogenic signalling. Covers both splice site and intronic mutations that cause exon 14 skipping.

Dose & Route

Oral · 450 mg once daily with food · Approved Feb 2021

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Ponvory

ponesimod

Selective S1P1 Receptor Modulator

Indication

Relapsing Multiple Sclerosis (RMS)

Mechanism

Highly selective S1P1 receptor modulator (no S1P3/4/5 off-target activity unlike fingolimod). Functional antagonism of S1P1 traps autoreactive CCR7+ lymphocytes in lymph nodes, reducing CNS-infiltrating lymphocyte traffic. Shorter half-life (~33 h) vs. fingolimod enables faster lymphocyte recovery after discontinuation — advantageous in infections or planned pregnancy. 14-day titration minimizes first-dose bradycardia.

Dose & Route

Oral · 20 mg once daily (after 14-day titration) · Approved Mar 2021

Qelbree

viloxazine ER

Selective NE Reuptake Inhibitor / 5-HT Modulator★ FIC (ADHD)

Indication

ADHD (6–17 years) — non-stimulant option

Mechanism

Selective norepinephrine reuptake inhibitor with additional 5-HT2B agonist and 5-HT2C antagonist activity — a serotonergic mechanism not shared by atomoxetine or guanfacine. The 5-HT interactions may modulate dopamine/norepinephrine release in PFC, contributing to ADHD symptom control. Non-stimulant; no scheduled substance classification; approved for pediatric use earlier than adult approval.

Dose & Route

Oral ER capsule · Once daily · Approved Apr 2021

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Skytrofa

lonapegsomatropin-tcgd

Once-Weekly Long-Acting GH

Indication

Pediatric Growth Hormone Deficiency — once-weekly SC injection

Mechanism

PEGylated recombinant hGH (somatropin) with a transient PEGylation strategy: growth hormone attached to PEG via an enzyme-labile linker that is gradually cleaved in vivo — releasing active somatropin over 1 week. Replaces daily GH injections with weekly dosing, reducing injection burden in children. GH stimulates IGF-1 production in liver → linear growth, protein synthesis, lipolysis; same physiologic mechanism as native GH.

Dose & Route

SC injection · Once weekly · Approved Aug 2021

Korsuva

difelikefalin

Peripheral κ-Opioid Receptor Agonist★ First-in-Class

Indication

Moderate-to-Severe Pruritus in CKD Patients on Hemodialysis

Mechanism

First peripheral κ-opioid receptor agonist for CKD pruritus. Selectively activates κ-opioid receptors on peripheral sensory nerve fibers and immune cells, attenuating itch signalling in uremic pruritus. Highly hydrophilic; does not cross the blood-brain barrier — avoiding centrally-mediated opioid side effects (sedation, abuse potential, dysphoria). Administered IV after each dialysis session 3× weekly.

Dose & Route

IV · 0.5 mcg/kg after each dialysis session (3×/week) · Approved Aug 2021

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Bylvay

odevixibat

IBAT Inhibitor★ First-in-Class

Indication

Pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC)

Mechanism

Inhibits the ileal bile acid transporter (IBAT/ASBT/SLC10A2), blocking the enterohepatic recirculation of bile acids by preventing their reabsorption from the terminal ileum. In PFIC, bile acid accumulation in hepatocytes → cholestasis → pruritus (via peripheral MRGPRx4/TGR5 activation by bile acid metabolites). Reducing bile acid pool size and serum bile acids → decreased pruritus. Non-systemic; acts locally in the gut.

Dose & Route

Oral · Once daily · Approved Jul 2021

Pylarify

piflufolastat F18

PSMA-Targeted PET Tracer

Indication

Prostate Cancer — PSMA-targeted PET imaging for staging and recurrence

Mechanism

18F-labeled PSMA-targeting small molecule (DCFPyL scaffold) for PET imaging. Binds PSMA (prostate-specific membrane antigen/FOLH1) overexpressed on prostate cancer cells. ¹⁸F label (t½ 110 min) enables central pharmacy distribution unlike ¹⁶⁸Ga tracers requiring on-site cyclotron. Standard uptake values in PSMA+ lesions allow accurate staging, biochemical recurrence localization, and patient selection for PSMA-targeted therapies (lutetium-177 PSMA-617).

Dose & Route

IV · Single dose before PET scan · Approved May 2021

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Fexinidazole

fexinidazole

Nitroimidazole Antiparasitic★ First-in-Class

Indication

Human African Trypanosomiasis (HAT) — sleeping sickness (T. brucei gambiense, stages 1 & 2)

Mechanism

First oral treatment effective for both stages of sleeping sickness. Prodrug reduced by nitroreductases inside T. brucei to highly reactive nitro radical intermediates that damage trypanosomal DNA and other macromolecules. Active against both Stage 1 (hemolymphatic) and Stage 2 (CNS-penetrant, previously requiring IV melarsoprol/eflornithine) — replacing complex hospital-based parenteral regimens with a 10-day oral course. Driven by Drugs for Neglected Diseases initiative (DNDi).

Dose & Route

Oral · Once daily for 10 days (with food) · Approved Jun 2021

Rylaze

asparaginase erwinia chrysanthemi (recombinant)-rywn

Recombinant Asparaginase

Indication

ALL/LBL — patients hypersensitive to E. coli-derived asparaginase

Mechanism

Recombinant form of Erwinia chrysanthemi asparaginase produced in Pseudomonas fluorescens. Asparaginase depletes circulating asparagine by hydrolyzing it to aspartate + NH3 — exploiting the dependence of leukemic lymphoblasts (which lack asparagine synthetase) on exogenous asparagine for protein synthesis and survival. Erwinia-derived enzyme is immunologically distinct from E. coli-derived asparaginase — used when E. coli asparaginase causes hypersensitivity reactions, without cross-reactivity.

Dose & Route

IM/IV · Monday-Wednesday-Friday schedule · Approved Jun 2021

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Brexafemme

ibrexafungerp

Triterpenoid Glucan Synthase Inhibitor★ First-in-Class

Indication

Vulvovaginal Candidiasis (VVC) — oral treatment

Mechanism

First oral non-azole antifungal for VVC. Triterpenoid derivative of enfumafungin that inhibits (1,3)-β-D-glucan synthase — same target as echinocandins but a structurally different (non-echinocandin) scaffold. Oral bioavailability enables systemic delivery unlike echinocandins (IV-only). Active against azole-resistant Candida (including C. auris and fluconazole-resistant C. glabrata). Two-dose oral regimen (same day).

Dose & Route

Oral · 300 mg twice in one day · Approved Jun 2021

Lybalvi

olanzapine / samidorphan

D2 Antagonist + Peripheral Opioid Antagonist★ First-in-Class

Indication

Schizophrenia · Bipolar I Disorder

Mechanism

Fixed-dose combination of olanzapine (D2/5-HT2A antagonist antipsychotic) with samidorphan, a μ-opioid receptor antagonist. Olanzapine causes weight gain and metabolic syndrome partly via opioid receptor-mediated central appetite stimulation; samidorphan counteracts this pathway. The combination preserves olanzapine's antipsychotic efficacy while attenuating weight gain — addressing the primary barrier to long-term olanzapine use.

Dose & Route

Oral · Once daily · Approved May 2021

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Truseltiq

infigratinib

FGFR1/2/3 TKI

Indication

FGFR2 Fusion/Rearrangement+ Cholangiocarcinoma — previously treated

Mechanism

Selective pan-FGFR1/2/3 kinase inhibitor targeting the ATP-binding site. FGFR2 gene fusions are found in ~10–15% of intrahepatic cholangiocarcinomas and drive constitutive RAS/MAPK and PI3K/STAT3 signalling. Infigratinib blocks FGFR2 kinase activity, suppressing tumor cell proliferation and survival in fusion-positive tumors. Approved alongside pemigatinib as second oral FGFR2 inhibitor for this rare cholangiocarcinoma subset.

Dose & Route

Oral · 125 mg once daily (21 days on/7 days off) · Approved May 2021

Nextstellis

drospirenone / estetrol

Oral Contraceptive (Native Estrogen)★ FIC (estetrol OCP)

Indication

Oral Contraception

Mechanism

First OCP containing estetrol (E4) — a human-specific native estrogen. Estetrol is produced only by the fetal liver during pregnancy; it acts as a selective estrogen receptor modulator — full ERα agonist in pituitary/hypothalamus (suppressing FSH/LH via negative feedback) but ERα antagonist/neutral in breast and vascular endothelium. Paired with drospirenone (progestin). Potentially lower VTE and breast cancer risk vs. EE-based OCPs due to E4's tissue-selective ER profile.

Dose & Route

Oral · 28-day cycle · Approved Apr 2021

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Zegalogue

dasiglucagon

Aqueous Glucagon Analog

Indication

Severe Hypoglycemia in Diabetes (≥6 years) — emergency rescue

Mechanism

Stabilized glucagon analog (His→Ala substitution at position 1) with aqueous solubility enabling a pre-filled, ready-to-inject SC solution that doesn't require reconstitution. Activates glucagon receptor in hepatocytes → adenylyl cyclase → cAMP → phosphorylase kinase → glycogenolysis + gluconeogenesis → rapid rise in blood glucose. Overcomes the reconstitution complexity of lyophilized glucagon kits, enabling emergency use by family members/caregivers.

Dose & Route

SC auto-injector · 0.6 mg single dose · Approved Mar 2021

Fotivda

tivozanib

VEGFR1/2/3 TKI (Highly Selective)

Indication

Relapsed/Refractory RCC — following ≥2 prior systemic therapies

Mechanism

Highly selective inhibitor of all three VEGFR kinases (VEGFR1/2/3) with minimal off-target activity against PDGFR/FGFR/KIT — resulting in a cleaner toxicity profile than sunitinib/sorafenib. 3-week on/1-week off dosing. Approved for later-line RCC specifically on the basis of TIVO-3 trial PFS benefit in patients who had received multiple prior VEGFR TKIs/immunotherapy, demonstrating activity even after prior anti-angiogenic failure.

Dose & Route

Oral · 1.34 mg once daily (3 weeks on / 1 week off) · Approved Mar 2021

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Nulibry

fosdenopterin

cPMP Substrate Replacement★ First-in-Class

Indication

Molybdenum Cofactor Deficiency Type A (MOCD-A) — rare neonatal metabolic disease

Mechanism

First treatment for MOCD Type A. MOCD-A is caused by MOCS1 mutations blocking synthesis of cyclic pyranopterin monophosphate (cPMP), a precursor in molybdenum cofactor (MoCo) biosynthesis. Without MoCo, sulfite oxidase/xanthine dehydrogenase/aldehyde oxidase are inactive → sulfite accumulation → toxic encephalopathy within days of birth. Fosdenopterin (synthetic cPMP analog) replaces the missing metabolite, restoring MoCo biosynthesis and preventing sulfite-induced neurological damage.

Dose & Route

IV · Daily · Approved Feb 2021

Aduhelm

aducanumab-avwa

Anti-Amyloid Plaque mAb

Indication

Alzheimer Disease — early symptomatic (amyloid PET-confirmed)

Mechanism

Human IgG1 mAb targeting aggregated amyloid-β, with preference for soluble protofibrils and insoluble plaques. Approved via accelerated approval pathway based on amyloid clearance on PET imaging (surrogate endpoint) rather than proven clinical benefit — generating significant controversy as confirmatory clinical trial data were inconsistent. Withdrawn from the EU market; limited CMS coverage in the US; superseded clinically by lecanemab (Leqembi) which showed actual cognitive slowing.

Dose & Route

IV infusion · Every 4 weeks · Accelerated approval Jun 2021