FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 955 total
Drug information is sourced from FDA labels and prescribing information. Always verify current prescribing information with your pharmacist or prescriber before starting, stopping, or changing any medication.
Phos-Lo
Calcium acetate
Fresenius Medical Care North America
1 INDICATIONS AND USAGE Calcium acetate is a phosphate binder indicated to reduce serum phosphorus in patients with end stage renal disease (ESRD). Ca…
Xywav
calcium, magnesium, potassium, and sodium oxybates
Jazz Pharmaceuticals Ireland Limited
1 INDICATIONS AND USAGE XYWAV is a central nervous system depressant indicated for the treatment of: • Cataplexy or excessive daytime sleepiness (EDS)…
Infasurf
calfactant
ONY Biotech Inc.
1 INDICATIONS AND USAGE INFASURF is indicated: to reduce the risk of respiratory distress syndrome (RDS) in preterm neonates <29 weeks of gestational …
ILARIS
canakinumab
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE ILARIS is an interleukin-1β blocker indicated for the treatment of: • Periodic Fever Syndromes ( 1.1 ): - Cryopyrin-Associated…
Epidiolex
CANNABIDIOL
Jazz Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE EPIDIOLEX is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or t…
Cablivi
CAPLACIZUMAB
Genzyme Corporation
1 INDICATIONS AND USAGE CABLIVI is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thro…
Qutenza
capsaicin
Averitas Pharma, Inc.
1 INDICATIONS AND USAGE QUTENZA is indicated in adults for the treatment of neuropathic pain associated with postherpetic neuralgia (PHN) and for neur…
CARBAMAZEPINE
CARBAMAZEPINE
Alembic Pharmaceuticals Inc.
INDICATIONS AND USAGE Epilepsy Carbamazepine is indicated for use as an anticonvulsant drug. Evidence supporting efficacy of carbamazepine as an antic…
DUOPA
CARBIDOPA AND LEVODOPA
AbbVie Inc.
1 INDICATIONS AND USAGE DUOPA ® is indicated for the treatment of motor fluctuations in patients with advanced Parkinson’s disease. DUOPA is a combina…
DHIVY
CARBIDOPA LEVODOPA
Avion Pharmaceuticals, LLC
1 INDICATIONS AND USAGE DHIVY is indicated for the treatment of Parkinson’s disease, post-encephalitic parkinsonism, and symptomatic parkinsonism that…
CARBIDOPA, LEVODOPA, AND ENTACAPONE
CARBIDOPA, LEVODOPA, AND ENTACAPONE
Sandoz Inc
1 INDICATIONS AND USAGE Carbidopa, levodopa and entacapone tablets, are indicated for the treatment of Parkinson’s disease. Carbidopa, levodopa and en…
Kyprolis
carfilzomib
Onyx Therapeutics, Inc.
1 INDICATIONS AND USAGE Kyprolis is a proteasome inhibitor that is indicated: for the treatment of adult patients with relapsed or refractory multiple…
Carbaglu
CARGLUMIC ACID
Recordati Rare Diseases
1. INDICATIONS AND USAGE CARBAGLU is a carbamoyl phosphate synthetase 1 (CPS 1) activator indicated in pediatric and adult patients as: Adjunctive the…
AMONDYS 45
CASIMERSEN
Sarepta Therapeutics, Inc.
1 INDICATIONS AND USAGE AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of th…
Inqovi
CEDAZURIDINE AND DECITABINE
Taiho Pharmaceutical Co., Ltd.
1 INDICATIONS AND USAGE INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and unt…
Oxervate
cenegermin-bkbj
Dompe S.p.A.
1 INDICATIONS AND USAGE OXERVATE ® (cenegermin-bkbj) ophthalmic solution 0.002% is indicated for the treatment of neurotrophic keratitis. OXERVATE is …
Anascorp
Centruroides immune F(ab)2
Rare Disease Therapeutics, Inc.
Treatment of clinical signs of scorpion envenomation…
ZYKADIA
ceritinib
Novartis Pharmaceuticals Corp.
1 INDICATIONS AND USAGE ZYKADIA ® is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are…
Brineura
CERLIPONASE ALFA
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE BRINEURA is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 di…
ZYRTEC
CETIRIZINE HYDROCHLORIDE
Kenvue Brands LLC
Uses temporarily relieves these symptoms due to hay fever or other upper respiratory allergies: runny nose sneezing itchy, watery eyes itching of the …
Erbitux
cetuximab
ImClone Systems Incorporated
1 INDICATIONS AND USAGE ERBITUX ® is an epidermal growth factor receptor (EGFR) antagonist indicated for treatment of: Head and Neck Cancer Locally or…
Chenix
chenodiol
Solvay
1 INDICATIONS AND USAGE CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. CTEXLI is a bile acid indicated for t…
Cholbam
CHOLIC ACID
Mirum Pharmaceuticals Inc.
1 INDICATIONS AND USAGE CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). ( 1.1 …
Carvykti
ciltacabtagene autoleucel
Janssen Research & Development, LLC
1 INDICATIONS AND USAGE CARVYKTI is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at l…
Cimetidine
CIMETIDINE
Chartwell RX, LLC
INDICATIONS AND USAGE Cimetidine tablets are indicated in: 1. Short-term treatment of active duodenal ulcer. Most patients heal within 4 weeks and the…
CINACALCET
CINACALCET
Aurobindo Pharma Limited
1 INDICATIONS AND USAGE Cinacalcet is a positive modulator of the calcium sensing receptor indicated for: Secondary Hyperparathyroidism (HPT) in adult…
POMBILITI ATGA
CIPAGLUCOSIDASE ALFA-ATGA
AMICUS THERAPEUTICS US, LLC
1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysoso…
Pombiliti and Opfolda
cipaglucosidase alfa-atga and miglustat
Amicus Therapeutics, Inc.
in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) w…