Rare Disease Drugs

131 FDA-approved drugs

TheraRadar tracks 131 FDA-approved rare disease drugs from 81 companies across 32 drug classes, led by Sanofi, with 436 active clinical trials in the pipeline. Explore recent approvals, top companies, active trials by indication, and drug classes for Rare Disease.

Rare Disease Approvals by Year

3

'15

2

'16

8

'17

8

'18

9

'19

7

'20

8

'21

10

'22

9

'23

8

'24

12

'25

2

'26

Recent Rare Disease Drug Approvals

TIVIDENOFUSP ALFA-EKNM

neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated ...

DENALI THERAPEUTICS INC.

COPPER HISTIDINATE

Menkes disease in pediatric patients...

SENTYNL THERAPS INC

DICHLORPHENAMIDE

DICHLORPHENAMIDE

primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variant...

thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset o...

ELAMIPRETIDE HYDROCHLORIDE

FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrom...

STEALTH BIOTHERAPS

DAWNZERA (AUTOINJECTOR)

DONIDALORSEN SODIUM

IONIS PHARMS INC

hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapteri...

acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older...

Duchenne muscular dystrophy (DMD) in patients 5 years of age and older...

SAPROPTERIN DIHYDROCHLORIDE

Zelvysia is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one...

Duchenne muscular dystrophy (DMD) in patients 5 years of age and older...

ZYDUS LIFESCIENCES

FITUSIRAN SODIUM

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...

DIAZOXIDE CHOLINE

hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PW...

cerebrotendinous xanthomatosis (CTX) in adults...

CONCIZUMAB-MTCI

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...

MARSTACIMAB-HNCQ

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...

FACTOR XATHROMBIN

ARIMOCLOMOL CITRATE

use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick d...

1 INDICATIONS & USAGE L-glutamine is indicated to reduce the acute complications of sickle cell dise...

XOLREMDI is indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglob...

XROMI is indicated to reduce the frequency of painful crises and reduce the need for blood transfusi...

Showing the 20 most recent of 131 rare disease drugs. View all 131 →

Top Rare Disease Companies

Sanofi 8 BIOMARIN PHARM 5 FERRING PHARMS INC 5 Takeda 4 LEADIANT BIOSCI INC 4 SENTYNL THERAPS INC 3 AMICUS THERAP US 3 NOVITIUM PHARMA 3 Sarepta Therapeutics 3 ALNYLAM PHARMS INC 3

Rare Disease Active Trials by Indication

436 active trials — the forward pipeline, not just approvals.

Immune Thrombocytopenia 149 Sickle Cell Disease 109 Hemophilia 73 Duchenne Muscular Dystrophy 49 Spinal Muscular Atrophy 23 Fabry Disease 14 Gaucher Disease 9 Mastocytosis 8

All competitive landscapes →

Rare Disease Drug Classes

Glucosylceramide Synthase Inhibitor 5

Corticosteroid 4

4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor 4

Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme 4

Carbonic Anhydrase Inhibitor 3

Plasma Kallikrein Inhibitor 3

Hydrolytic Lysosomal Glycogen-specific Enzyme 3

Antisense Oligonucleotide 3

Hydrolytic Lysosomal Glucocerebroside-specific Enzyme 3

Bile Acid 2

Rare Disease at a Glance

Total Drugs: 131
Companies: 81
Drug Classes: 32
Active Trials: 436
Latest Approval: 2026-03-24