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.
Cerdelga
ELIGLUSTAT
Genzyme Corporation
1 INDICATIONS AND USAGE CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive…
Skysona
ELIVALDOGENE AUTOTEMCEL
Genetix Biotherapeutics Inc.
1 INDICATIONS AND USAGE SKYSONA is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral a…
VIMIZIM
ELOSULFASE ALFA
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). VIMIZIM…
Empliciti
elotuzumab
Bristol-Myers Squibb Co.
1 INDICATIONS AND USAGE • EMPLICITI is indicated in combination with lenalidomide and dexamethasone for the treatment of adult patients with multiple …
ELREXFIO
ELRANATAMAB-BCMM
U.S. Pharmaceuticals
1 INDICATIONS AND USAGE ELREXFIO is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at le…
ELTROMBOPAG
ELTROMBOPAG
Dr. Reddy's Laboratories Inc.,
1 INDICATIONS AND USAGE Eltrombopag is a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediatric patie…
Eltrombopag
ELTROMBOPAG OLAMINE
Novadoz Pharmaceuticals LLC
1 INDICATIONS AND USAGE Eltrombopag tablets are a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediat…
GAMIFANT
EMAPALUMAB-LZSG
Swedish Orphan Biovitrum AB (publ)
1 INDICATIONS AND USAGE GAMIFANT is indicated for the treatment of: adult and pediatric (newborn and older) patients with primary hemophagocytic lymph…
Hemlibra
EMICIZUMAB
Genentech, Inc.
1 INDICATIONS AND USAGE HEMLIBRA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p…
Descovy
emtricitabine and tenofovir alafenamide
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE HIV-1 Treatment ( 1.1 ): DESCOVY is a two-drug combination of emtricitabine (FTC) and tenofovir alafenamide (TAF), both HIV nu…
Idhifa
enasidenib
Celgene Corporation
1 INDICATIONS AND USAGE IDHIFA is an isocitrate dehydrogenase-2 inhibitor indicated for the treatment of adult patients with relapsed or refractory ac…
Braftovi
encorafenib
Array BioPharma Inc. (a wholly owned subsidiary of Pfizer Inc.)
1 INDICATIONS AND USAGE BRAFTOVI is a kinase inhibitor indicated: Melanoma • in combination with binimetinib, for the treatment of patients with unres…
ROZLYTREK
entrectinib
Genentech, Inc.
1 INDICATIONS AND USAGE ROZLYTREK is a kinase inhibitor indicated for the treatment of: Adult patients with ROS1- positive metastatic non-small cell l…
Epkinly
epcoritamab-bysp
Genmab US, Inc.
1 INDICATIONS AND USAGE EPKINLY is a bispecific CD20-directed CD3 T-cell engager indicated: For the treatment of adult patients with relapsed or refra…
EPIPEN
EPINEPHRINE
Viatris Specialty LLC
1 INDICATIONS AND USAGE EpiPen are indicated in the emergency treatment of allergic reactions (Type I) including anaphylaxis to stinging insects (e.g.…
Ellence
Epirubicin
Pharmacia & Upjohn Company
1 INDICATIONS AND USAGE ELLENCE is indicated as a component of adjuvant therapy in patients with evidence of axillary node tumor involvement following…
WAINUA
EPLONTERSEN
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAINUA…
Epogen
Epoetin alfa
Amgen, Inc.
1 INDICATIONS AND USAGE Epogen is an erythropoiesis-stimulating agent (ESA) indicated for: Treatment of anemia due to: - Chronic Kidney Disease (CKD) …
Epoprostenol
EPOPROSTENOL
Mylan Institutional LLC
1 INDICATIONS AND USAGE Epoprostenol for injection is indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) to improve ex…
Flolan
EPOPROSTENOL SODIUM
GlaxoSmithKline LLC
1 INDICATIONS AND USAGE FLOLAN is indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group I) to improve exercise capacity. Tri…
Halaven
eribulin mesylate
Eisai Inc.
1 INDICATIONS AND USAGE HALAVEN is a microtubule inhibitor indicated for the treatment of patients with: Metastatic breast cancer who have previously …
Erwinase
Erwinia L-asparaginase
Jazz Pharmaceuticals, Inc.
Treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.…
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
MARKSANS PHARMA LIMITED
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
MARKSANS PHARMA LIMITED
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
ESOMEPRAZOLE MAGNESIUM DELAYED-RELEASE CAPSULES, 20 MG
MARKSANS PHARMA LIMITED
ESTRADIOL / NORETHINDRONE ACETATE
ESTRADIOL AND NORETHINDRONE ACETATE
Breckenridge Pharmaceutical, Inc.
Enbrel
etanercept
Immunex Corporation
1 INDICATIONS AND USAGE Enbrel is a tumor necrosis factor (TNF) blocker indicated for the treatment of: Adult patients with: Rheumatoid Arthritis (RA)…
Exondys 51
ETEPLIRSEN
Sarepta Therapeutics, Inc.
1 INDICATIONS AND USAGE EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of th…