FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 968 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.
Tryngolza
olezarsen
Ionis Pharmaceuticals
1 INDICATIONS AND USAGE TRYNGOLZA is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). T…
XENPOZYME
OLIPUDASE ALFA-RPCP
Genzyme Corporation
1 INDICATIONS AND USAGE XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) i…
OLMESARTAN MEDOXOMIL AND HYDROCHLOROTHIAZIDE
OLMESARTAN MEDOXOMIL AND HYDROCHLOROTHIAZIDE
Ascend Laboratories, LLC
Rezlidhia
olutasidenib
Rigel Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Relapsed or Refractory Acute Myeloid Leukemia REZLIDHIA is indicated for the treatment of adult patients with relapsed or refr…
Synribo
omacetaxine mepesuccinate
IVAX International GmbH
Treatment of adult patients with chronic or accelerated phase chronic myeloid leukemia (CML) with resistance and/or intolerance to two or more tyrosin…
Skyclarys
omaveloxolone
Biogen U.S. Corporation
1 INDICATIONS AND USAGE SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. SKYCLARYS i…
Omeprazole
OMEPRAZOLE
PD-Rx Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Omeprazole is a proton pump inhibitor (PPI) indicated for the: • Treatment of active duodenal ulcer in adults (1.1) • Eradicat…
Omisirge
OMIDUBICEL-ONLV
Gamida Cell Inc.
1 INDICATIONS AND USAGE OMISIRGE is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for the…
ITVISMA
ONASEMNOGENE ABEPARVOVEC-BRVE
Novartis Gene Therapies, Inc.
1 INDICATIONS AND USAGE ITVISMA is indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and old…
Zolgensma
ONASEMNOGENE ABEPARVOVEC-XIOI
Novartis Gene Therapies, Inc.
1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less …
Isturisa
osilodrostat
Recordati Rare Diseases, Inc
1 INDICATIONS AND USAGE ISTURISA is indicated for the treatment of endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is …
Tagrisso
osimertinib
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE TAGRISSO is a kinase inhibitor indicated for: • adjuvant therapy after tumor resection in adult patients with non-small cell l…
OXALIPLATIN
OXALIPLATIN
Accord Healthcare Inc.
1 INDICATIONS AND USAGE Oxaliplatin Injection, in combination with infusional fluorouracil and leucovorin, is indicated for: adjuvant treatment of sta…
Taxol
Paclitaxel
Bristol-Myers Squibb Pharmaceutical Research Institute
INDICATIONS AND USAGE Paclitaxel Injection, USP is indicated as subsequent therapy for the treatment of advanced carcinoma of the ovary. As first-line…
Abraxane
paclitaxel protein-bound particles
Abraxis BioScience, LLC
1 INDICATIONS AND USAGE ABRAXANE is a microtubule inhibitor indicated for the treatment of: • Metastatic breast cancer, after failure of combination c…
Vonjo
PACRITINIB
Sobi, Inc.
1 INDICATIONS AND USAGE VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or …
Cytalux
pafolacianine
On Target Laboratories, LLC
1. INDICATIONS AND USAGE CYTALUX is indicated as an adjunct for intraoperative identification of: Malignant lesions in adult patients with ovarian can…
Yorvipath
palopegteriparatide
Ascendis Pharma Bone Diseases A/S
1 INDICATIONS AND USAGE YORVIPATH is indicated for the treatment of hypoparathyroidism in adults. YORVIPATH is a parathyroid hormone analog (PTH(1-34)…
Sohonos
PALOVAROTENE
Ipsen Biopharmaceuticals, Inc.
1 INDICATIONS AND USAGE SOHONOS is indicated for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years…
Palsonify
paltusotine
Crinetics Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE PALSONIFY is indicated for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom s…
Farydak
panobinostat
zr pharma& GmbH
FARYDAK (panobinostat) in combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma who have recei…
Natpara
parathyroid hormone
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE NATPARA is a parathyroid hormone indicated as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hyp…
Zemplar
paricalcitol
Abbvie, inc.
1 INDICATIONS AND USAGE ZEMPLAR is a vitamin D analog indicated in adults and pediatric patients 10 years and older for the prevention and treatment o…
Signifor
pasireotide
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE SIGNIFOR is a somatostatin analog indicated for the treatment of adult patients with Cushing's disease for whom pituitary surg…
Onpattro
PATISIRAN
Alnylam Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ONPA…