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.
Ethamolin
Ethanolamine oleate
QOL Medical
INDICATIONS AND USAGE ETHAMOLIN Injection is indicated for the treatment of patients with esophageal varices that have recently bled, to prevent reble…
Lipiodol
ethiodized oil injection
Guerbet LLC
1 INDICATIONS AND USAGE Lipiodol is an oil-based radio-opaque contrast agent indicated for: hysterosalpingography in adults lymphography in adult and …
HEMGENIX
ETRANACOGENE DEZAPARVOVEC
CSL Behring
1 INDICATIONS AND USAGE HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for treatment of adults with Hemophilia B (congenita…
Waskyra
etuvetidigene autotemcel
Fondazione Telethon ETS
treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for who…
Everolimus
EVEROLIMUS
Breckenridge Pharmaceutical, Inc.
1. INDICATIONS AND USAGE Everolimus tablets are a kinase inhibitor indicated for the treatment of: Postmenopausal women with advanced hormone receptor…
Evkeeza
evinacumab-dgnb
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE EVKEEZA is indicated as an adjunct to diet and exercise and other low-density lipoprotein-cholesterol (LDL-C) lowering therapi…
Repatha
evolocumab
Amgen Inc.
1 INDICATIONS AND USAGE REPATHA is indicated: To reduce the risk of major adverse cardiovascular (CV) events (CV death, myocardial infarction, stroke,…
CASGEVY
EXAGAMGLOGENE AUTOTEMCEL
Vertex Pharmaceuticals Incorporated
1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vas…
Aromasin
Exemestane
Pharmacia & Upjohn
1 INDICATIONS AND USAGE AROMASIN is an aromatase inhibitor indicated for: • adjuvant treatment of postmenopausal women with estrogen-receptor positive…
Corifact
factor XIII concentrate (human)
CSL Behring LLC
For the routine prophylactic treatment of congenital factor XIII deficiency ||| Indicated for routine prophylactic treatment and peri-operative manage…
Enhertu
fam-trastuzumab deruxtecan-nxki
Daiichi Sankyo, Inc.
1 INDICATIONS AND USAGE ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated: HER2-Positive Metastatic Breast Cancer in…
Vowst
fecal microbiota spores, live-brpk
Aimmune Therapeutics, Inc.
1 INDICATIONS AND USAGE VOWST is indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and ol…
Rebyota
fecal microbiota, live - jslm
Ferring Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE REBYOTA is indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of a…
Inrebic
FEDRATINIB HYDROCHLORIDE
Celgene Corporation
1 INDICATIONS AND USAGE INREBIC ® is indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycyt…
Felbamate
FELBAMATE
Taro Pharmaceuticals U.S.A., Inc.
INDICATIONS AND USAGE Felbamate oral suspension, USP is not indicated as a first line antiepileptic treatment (see Warnings ). Felbamate oral suspensi…
Fintepla
fenfluramine
UCB, Inc.
1 INDICATIONS AND USAGE FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in …
Ferabright
ferumoxytol
Covis Pharma GmbH
1 INDICATIONS AND USAGE FERABRIGHT is indicated for magnetic resonance imaging (MRI) of the brain in adults with known or suspected malignant neoplasm…
Beqvez
fidanacogene elaparvovec-dzkt
Pfizer Inc.
treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who: Currently use factor IX prophylaxis therapy, or; Have …
Dificid
fidaxomicin
Cubist Pharmaceuticals LLC, a subsidiary of Merck Sharp & Dohme LLC
1 INDICATIONS AND USAGE DIFICID is a macrolide antibacterial indicated in adult and pediatric patients 6 months of age and older for the treatment of …
Neupogen
filgrastim
Amgen, Inc.
1 INDICATIONS AND USAGE NEUPOGEN is a leukocyte growth factor indicated to Decrease the incidence of infection‚ as manifested by febrile neutropenia‚ …
Omegaven
fish oil triglycerides
Fresenius Kabi USA, LLC
1 INDICATIONS AND USAGE Omegaven is indicated as a source of calories and fatty acids in pediatric patients with parenteral nutrition-associated chole…
Qfitlia
FITUSIRAN
Genzyme Corporation
1 INDICATIONS AND USAGE QFITLIA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric pa…
Fludara
Fludarabine phosphate
Berlex Laboratories, Inc.
1 INDICATIONS AND USAGE Fludarabine Phosphate Injection is a nucleotide metabolic inhibitor indicated for: The treatment of adult patients with B-cell…
Retisert
Fluocinolone
Bausch & Lomb Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE RETISERT ® is indicated for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye. RETISE…
Gonal-F
Follitropin alfa, recombinant
EMD Serono, Inc.
1 INDICATIONS AND USAGE GONAL-f is indicated for: GONAL-f is a gonadotropin indicated for: Women: Induction of ovulation and pregnancy in oligo-anovul…
Antizole
fomepizole
Par Pharmaceuticals, Inc
Use for suspected or confirmed methanol poisoning, either alone or in combination with hemodialysis ||| As an antidote to ethylene glycol (antifreeze)…
VYALEV
FOSCARBIDOPA/FOSLEVODOPA
AbbVie Inc.
1 INDICATIONS AND USAGE VYALEV is indicated for the treatment of motor fluctuations in adults with advanced Parkinson’s disease (PD). VYALEV is a comb…