Rare Disease Drugs
130 FDA-approved drugs
Recent Approvals
AVLAYAH
TIVIDENOFUSP ALFA-EKNM
neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated ...
2026-03-24
DENALI THERAPEUTICS INC.
ZYCUBO
COPPER HISTIDINATE
Menkes disease in pediatric patients...
2026-01-12
SENTYNL THERAPS INC
DICHLORPHENAMIDE
DICHLORPHENAMIDE
primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variant...
2025-11-18
RISING
KYGEVVI
DOXECITINE
thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset o...
2025-11-03
UCB INC
FORZINITY
ELAMIPRETIDE HYDROCHLORIDE
FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrom...
2025-09-19
STEALTH BIOTHERAPS
DAWNZERA (AUTOINJECTOR)
DONIDALORSEN SODIUM
2025-08-21
IONIS PHARMS INC
SEPHIENCE
SEPIAPTERIN
hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapteri...
2025-07-28
PTC THERAP
EKTERLY
SEBETRALSTAT
acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older...
2025-07-03
KALVISTA
PYQUVI
DEFLAZACORT
Duchenne muscular dystrophy (DMD) in patients 5 years of age and older...
2025-06-30
AUCTA
ZELVYSIA
SAPROPTERIN DIHYDROCHLORIDE
Zelvysia is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one...
2025-04-29
AUCTA
JAYTHARI
DEFLAZACORT
Duchenne muscular dystrophy (DMD) in patients 5 years of age and older...
2025-04-08
ZYDUS LIFESCIENCES
QFITLIA
FITUSIRAN SODIUM
routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...
2025-03-28
Sanofi
VYKAT XR
DIAZOXIDE CHOLINE
hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PW...
2025-03-26
SOLENO THERAP
CTEXLI
CHENODIOL
cerebrotendinous xanthomatosis (CTX) in adults...
2025-02-21
MIRUM
ALHEMO
CONCIZUMAB-MTCI
routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...
2024-12-20
Novo Nordisk
THROMBIN
HYMPAVZI
MARSTACIMAB-HNCQ
routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p...
2024-10-11
Pfizer
FACTOR XATHROMBIN
MIPLYFFA
ARIMOCLOMOL CITRATE
use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick d...
2024-09-20
ZEVRA DENMARK
L-GLUTAMINE
L-GLUTAMINE
1 INDICATIONS & USAGE L-glutamine is indicated to reduce the acute complications of sickle cell dise...
2024-07-08
NOVITIUM PHARMA
XOLREMDI
MAVORIXAFOR
XOLREMDI is indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglob...
2024-04-26
X4 PHARMS
XROMI
HYDROXYUREA
XROMI is indicated to reduce the frequency of painful crises and reduce the need for blood transfusi...
2024-04-04
NOVA LABS LTD
Showing 20 of 130 drugs
Top Companies
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
Quick Stats
- Total Drugs
- 130
- Companies
- 80
- Drug Classes
- 32
- Latest Approval
- 2026-03-24