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 155 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

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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

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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

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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

📋

#	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

💊

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

🔍

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

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

🔍

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

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

🔍

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

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