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.
Nulibry
fosdenopterin
Origin Biosciences, Inc.
1 INDICATIONS AND USAGE NULIBRY is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. NULIBRY is…
Cerebyx
Fosphenytoin
Warner-Lambert Company
1 INDICATIONS AND USAGE CEREBYX is indicated for the treatment of generalized tonic-clonic status epilepticus and prevention and treatment of seizures…
Lytgobi
futibatinib
Taiho Oncology, Inc.
Treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast…
Gralise
gabapentin
Golf Acquiror LLC
1 INDICATIONS AND USAGE GRALISE is indicated for the management of postherpetic neuralgia. GRALISE is not substitutable with other gabapentin products…
Horizant
gabapentin enacarbil
Arbor Pharmaceuticals, LLC
1 INDICATIONS AND USAGE HORIZANT is indicated for: treatment of moderate-to-severe primary Restless Legs Syndrome (RLS) in adults. ( 1.1 ) management …
NETSPOT
Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide
Advanced Accelerator Applications, USA
1 INDICATIONS AND USAGE NETSPOT, after radiolabeling with gallium-68, is indicated for use with positron emission tomography (PET) for localization of…
NAGLAZYME
GALSULFASE
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). NAGLAZYME has been shown …
Ztalmy
ganaxolone
Marinus Pharmaceuticals
1 INDICATIONS AND USAGE ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (…
Zirgan
ganciclovir
Sirion Therapeutics, Inc.
1 INDICATIONS AND USAGE ZIRGAN is indicated for the treatment of acute herpetic keratitis (dendritic ulcers) in adults and pediatric patients aged 2 y…
Vitrasert Implant
Ganciclovir intravitreal implant
Bausch & Lomb Surgical, Chiron Vision Products
Treatment of CMV retinitis in patients with acquired immunodeficiency syndrome.…
Cytovene
Ganciclovir sodium
Syntex (USA), Inc.
1 INDICATIONS AND USAGE Ganciclovir Injection Ganciclovir Injection is a deoxynucleoside analogue cytomegalovirus (CMV) DNA polymerase inhibitor indic…
ANDEMBRY
GARADACIMAB
CSL
1 INDICATIONS AND USAGE ANDEMBRY is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 1…
Iressa
gefitinib
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE IRESSA is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors h…
Mylotarg
Gemtuzumab ozogamicin
Wyeth Pharmaceuticals, Inc., a Pfizer Company
1 INDICATIONS AND USAGE MYLOTARG is a CD33-directed antibody and cytotoxic drug conjugate indicated for: • treatment of newly-diagnosed CD33-positive …
XOSPATA
gilteritinib
Astellas Pharma US, Inc.
1 INDICATIONS AND USAGE XOSPATA is a kinase inhibitor indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leuk…
DUVYZAT
GIVINOSTAT
Italfarmaco SPA
1 INDICATIONS AND USAGE DUVYZAT is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. DUVYZAT is a…
GIVLAARI
givosiran
Alnylam Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is an aminolevulinate synthase …
DAURISMO
Glasdegib
Pfizer, Inc.
1 INDICATIONS AND USAGE DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (A…
Copaxone
glatiramer acetate
Teva Pharmaceuticals USA
1 INDICATIONS AND USAGE COPAXONE is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome…
MAVYRET
glecaprevir and pibrentasvir
AbbVie, Inc.
1 INDICATIONS AND USAGE MAVYRET is indicated for the treatment of adult and pediatric patients 3 years and older with acute or chronic hepatitis C vir…
Voraxaze
glucarpidase
BTG International Inc.
1 INDICATIONS AND USAGE VORAXAZE is indicated to reduce toxic plasma methotrexate concentration (greater than 1 micromole per liter) in adult and pedi…
Endari
GLUTAMINE
Emmaus Medical, Inc.
1 INDICATIONS AND USAGE Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age an…