FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 964 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.
XEOMIN
INCOBOTULINUMTOXINA
Merz Pharmaceuticals, LLC
1 INDICATIONS AND USAGE XEOMIN is an acetylcholine release inhibitor and neuromuscular blocking agent indicated for the treatment or improvement of: C…
Uplizna
INEBILIZUMAB
Horizon Therapeutics USA, Inc.
1 INDICATIONS AND USAGE UPLIZNA is a CD19-directed cytolytic antibody indicated for the treatment of: Neuromyelitis optica spectrum disorder (NMOSD) i…
Truseltiq
infigratinib
QED Therapeutics, Inc.
Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2…
Remicade
infliximab
Janssen Biotech, Inc.
1 INDICATIONS AND USAGE REMICADE is a tumor necrosis factor (TNF) blocker indicated for: Crohn's Disease : reducing signs and symptoms and inducing an…
Tegsedi
inotersen
Ionis/AstraZeneca
Indicated for polyneuropathy of hATTR amyloidosis in adults.…
Besponsa
inotuzumab ozogamicin
Wyeth Pharmaceuticals, LLC
1. INDICATIONS AND USAGE BESPONSA is indicated for the treatment of relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia…
Intron A
Interferon alfa-2b (recombinant)
Schering Corporation
Treatment of selected patients with AIDS-related Kaposi's sarcoma.…
Avonex
Interferon beta-1a
Biogen, Inc.
1 INDICATIONS AND USAGE AVONEX is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, …
Betaseron
Interferon beta-1b
Chiron Corp. & Berlex Laboratories
1 INDICATIONS AND USAGE BETASERON is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrom…
Actimmune
Interferon gamma 1-b
Horizon Therapeutic Ireland DAC
1 INDICATIONS AND USAGE ACTIMMUNE is indicated for reducing the frequency and severity of serious infections associated with Chronic Granulomatous Dis…
CARNEXIV
intravenous carbamazepine
Lundbeck LLC
Replacement therapy for oral carbamazepine formulations, when oral administration is temporarily not feasible, in adults with the following seizure ty…
AdreView
Iobenguane I 123
GE Healthcare, Inc.
1 INDICATIONS AND USAGE AdreView is a radiopharmaceutical agent for gamma-scintigraphy indicated for: use in the detection of primary or metastatic ph…
Azedra Ultratrace
Iobenguane I 131
Progenics Pharmaceuticals, Inc.
AZEDRA is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced…
Yervoy
ipilimumab
Bristol-Myers Squibb Company
1 INDICATIONS AND USAGE YERVOY is a human cytotoxic T-lymphocyte antigen 4 (CTLA-4)-blocking antibody indicated for: Melanoma • Treatment of unresecta…
FABHALTA
IPTACOPAN
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (…
Onivyde
irinotecan liposome injection
Ipsen Biopharmaceuticals, Inc.
1 INDICATIONS AND USAGE ONIVYDE is indicated, in combination with oxaliplatin, fluorouracil and leucovorin for the first-line treatment of adult patie…
Radiogardase
iron(III)-hexacyanoferrate(II)
Heyl Chemisch-Pharmzeutische Fabrik GMBH & Co, KG
1 INDICATIONS AND USAGE Radiogardase is indicated for treatment of patients with known or suspected internal contamination with radioactive cesium and…
Sarclisa
isatuximab-irfc
Sanofi-Aventis U.S. LLC
1 INDICATIONS AND USAGE SARCLISA is indicated: in combination with pomalidomide and dexamethasone, for the treatment of adult patients with multiple m…
Cresemba
isavuconazonium sulfate
Astellas Pharma Global Development Inc.
1 INDICATIONS AND USAGE CRESEMBA ® is an azole antifungal indicated for the treatment of: Invasive aspergillosis ( 1.1 ) and Invasive mucormycosis ( 1…
CORLANOR
ivabradine
Amgen, Inc.,
1. INDICATIONS AND USAGE Corlanor (ivabradine) is a hyperpolarization-activated cyclic nucleotide-gated channel blocker indicated: To reduce the risk …
Kalydeco
IVACAFTOR
Vertex Pharmaceuticals Incorporated
1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutat…
Tibsovo
IVOSIDENIB
Servier Pharmaceutical LLC
1 INDICATIONS AND USAGE TIBSOVO is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for patients with a susceptible IDH1 mutation as detected …
Ninlaro
ixazomib citrate
Takeda Development Center Americas, Inc.
1 INDICATIONS AND USAGE NINLARO is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma wh…
IXIARO
Japanese encephalitis vaccine, inactivated, adsorbed
Intercell AG
IXIARO is a vaccine indicated for the prevention of disease caused by Japanese encephalitis virus (JEV). IXIARO is approved for use in individuals 2 m…
KETALAR
KETAMINE HYDROCHLORIDE
Par Health USA, LLC
1 INDICATIONS AND USAGE KETALAR (ketamine hydrochloride) injection is indicated: as the sole anesthetic agent for diagnostic and surgical procedures t…
L-Glutamine
L-GLUTAMINE
ANI Pharmaceuticals, Inc.
INDICATIONS For the temporary relief of mild abdominal pain or discomfort, diarrhea, minor joint pain, general weakness, muscle fatigue, sadness, or a…