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.
IOPIDINE 1%
APRACLONIDINE HYDROCHLORIDE OPHTHALMIC SOLUTION
Harrow Eye, LLC
IOPIDINE 1% Ophthalmic Solution is indicated to control or prevent post-surgical elevations in IOP that occur in patients after argon laser trabeculop…
OTEZLA�
apremilast
Amgen Inc.
1 INDICATIONS AND USAGE OTEZLA/OTEZLA XR, an inhibitor of phosphodiesterase 4 (PDE4), is indicated for the treatment of: Adult patients with: Active p…
MIPLYFFA
ARIMOCLOMOL CITRATE
Acer Therapeutics Inc.
1 INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick d…
Abilify
aripiprazole
Otsuka Pharmaceutical Development
1 INDICATIONS AND USAGE ABILIFY (aripiprazole) Tablets are indicated for the treatment of: Schizophrenia Acute Treatment of Manic and Mixed Episodes a…
Trisenox
Arsenic trioxide
Teva Branded Pharmaceutical Products R&D, Inc.
1 INDICATIONS AND USAGE TRISENOX is an arsenical indicated: In combination with tretinoin for treatment of adults with newly-diagnosed low-risk acute …
Coartem
artemether/lumefantrine
Novartis Pharmaceuticals
1 INDICATIONS AND USAGE Coartem Tablets are indicated for treatment of acute, uncomplicated malaria infections due to Plasmodium falciparum (P. falcip…
Scemblix
asciminib
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE SCEMBLIX is indicated for the treatment of adult patients with: Newly diagnosed Philadelphia chromosome-positive chronic myelo…
Ascor
ascorbic acid
McGuff Pharmaceuticals Inc.
1. INDICATIONS AND USAGE ASCOR is vitamin C indicated for the short term (up to 1 week) treatment of scurvy in adult and pediatric patients age 5 mont…
Strensiq
ASFOTASE ALFA
Alexion Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE STRENSIQ ® is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP). STR…
Rylaze
asparaginase erwinia chrysanthemi (recombinant)-rywn
Jazz Pharmaceuticals Ireland Limited
1 INDICATIONS AND USAGE RYLAZE is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia…
TECENTRIQ
atezolizumab
Genentech, Inc.
1 INDICATIONS AND USAGE TECENTRIQ is a programmed death-ligand 1 (PD-L1) blocking antibody indicated: Non-Small Cell Lung Cancer (NSCLC) as adjuvant t…
Tecentriq and Avastin
atezolizumab + bevacizumab
Genentech, Inc.
TECENTRIQ, in combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not receiv…
Lenmeldy
ATIDARSAGENE AUTOTEMCEL
Orchard Therapeutics (Europe) Ltd
1 INDICATIONS AND USAGE LENMELDY is indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile…
INMAZEB
atoltivimab, maftivimab, and odesivimab-ebgn
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE INMAZEB is indicated for the treatment of infection caused by Orthoebolavirus zairense in adult and pediatric patients, includ…
MEPRON
ATOVAQUONE
GlaxoSmithKline LLC
1 INDICATIONS AND USAGE MEPRON oral suspension is a quinone antimicrobial drug indicated for: • Prevention of Pneumocystis jirovecii pneumonia (PCP) i…
MALARONE
ATOVAQUONE AND PROGUANIL HYDROCHLORIDE
GlaxoSmithKline LLC
1 INDICATIONS AND USAGE MALARONE is an antimalarial indicated for: • prophylaxis of Plasmodium falciparum malaria, including in areas where chloroquin…
Vanrafia
atrasentan
Novartis
1 INDICATIONS AND USAGE VANRAFIA is indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid diseas…
Tavneos
avacopan
ChemoCentryx, Inc.
1 INDICATIONS AND USAGE TAVNEOS is indicated as an adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody …
Nexviazyme
AVALGLUCOSIDASE ALFA-NGPT
Genzyme Corporation
1 INDICATIONS AND USAGE NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid al…
Ayvakit
avapritinib
Blueprint Medicines Corporation
1 INDICATIONS AND USAGE AYVAKIT is a kinase inhibitor indicated for: Gastrointestinal Stromal Tumor (GIST) the treatment of adults with unresectable o…
Doptelet
avatrombopag
Sobi, Inc.
1 INDICATIONS AND USAGE DOPTELET is a thrombopoietin receptor agonist indicated for the treatment of: Thrombocytopenia in adult patients with chronic …
Bavencio
avelumab (Recombinant human monoclonal IgG1 antibody against programmed death ligand-1)
EMD Serono Research and Development Institute, Inc.
1 INDICATIONS AND USAGE BAVENCIO is a programmed death ligand-1 (PD-L1) blocking antibody indicated for: Merkel Cell Carcinoma (MCC) Adults and pediat…
Avmapki Fakzynja Co-Pack
avutometinib; defactinib
Verastem, Inc.
1 INDICATIONS AND USAGE AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS -mutated recurrent low-grade serous ovaria…
Niktimvo
axatilimab-csfr
Incyte Corporation
1 INDICATIONS AND USAGE NIKTIMVO is indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines…
Yescarta
axicabtagene ciloleucel
Kite Pharma, Inc.
1 INDICATIONS AND USAGE YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Adult patient…
Azacitidine
AZACITIDINE
Dr. Reddy's Laboratories Inc.
1 INDICATIONS AND USAGE Azacitidine for injection is a nucleoside metabolic inhibitor indicated for the treatment of: Adult patients with the followin…
Azacitidine
AZACITIDINE FOR
Pilnova Pharma Inc
1 INDICATIONS AND USAGE Azacitidine for injection is a nucleoside metabolic inhibitor indicated for the treatment of: Adult patients with the followin…
Azithromycin
AZITHROMYCIN
Preferred Pharmaceuticals Inc.
1 INDICATIONS AND USAGE Azithromycin is a macrolide antibacterial drug indicated for the treatment of patients with mild to moderate infections caused…