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.
Blincyto
blinatumomab
Amgen, Inc.
1 INDICATIONS AND USAGE BLINCYTO is a bispecific CD19-directed CD3 T-cell engager indicated for the treatment of adult and pediatric patients one mont…
Velcade
bortezomib
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE VELCADE is a proteasome inhibitor indicated for: treatment of adult patients with multiple myeloma ( 1.1 ) treatment of adult …
Bosentan
BOSENTAN
Actavis Pharma, Inc.
1 INDICATIONS AND USAGE Bosentan tablets are indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1): in adults to improve …
Bosulif
bosutinib
PF PRISM C.V.
1 INDICATIONS AND USAGE BOSULIF is indicated for the treatment of: • Adult and pediatric patients 1 year of age and older with chronic phase (CP) Phil…
Botox
botulinum toxin type A
Allergan, Inc.
1 INDICATIONS AND USAGE BOTOX is an acetylcholine release inhibitor and a neuromuscular blocking agent indicated for: Treatment of overactive bladder …
Myobloc
Botulinum toxin type B
Soltice Neurosciences, LLC
1 INDICATIONS AND USAGE MYOBLOC is an acetylcholine release inhibitor indicated for: Treatment of cervical dystonia to reduce the severity of abnormal…
BabyBIG
Botulism immune globulin
California Department of Public Health
1 INDICATIONS AND USAGE BabyBIG ® , Botulism Immune Globulin Intravenous (Human), is indicated for the treatment of infant botulism caused by toxin ty…
Adcetris
brentuximab vedotin
Seagen Inc.
1 INDICATIONS AND USAGE ADCETRIS is a CD30-directed antibody and microtubule inhibitor conjugate indicated for treatment of: • Adult patients with pre…
TECARTUS�
brexucabtagene autoleucel
Kite Pharma, Inc.
1 INDICATIONS AND USAGE TECARTUS is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: TECARTUS is a…
Alunbrig
brigatinib
Takeda Development Center Americas, Inc.
1 INDICATIONS AND USAGE ALUNBRIG is indicated for the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small …
DORC ILM-Blue
Brilliant Blue G Ophthalmic Solution 0.025%
Dutch Ophthalmic Research Center International BV
TissueBlue (Brilliant Blue G Ophthalmic Solution) 0.025% is indicated to selectively stain the internal limiting membrane (ILM).…
Tembexa
brincidofovir
Emergent BioDefense Operations Lansing LLC
1 INDICATIONS AND USAGE TEMBEXA is an orthopoxvirus nucleotide analog DNA polymerase inhibitor and is indicated for the treatment of human smallpox di…
Eohilia
budesonide
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE EOHILIA ™ is indicated for 12 weeks of treatment in adult and pediatric patients 11 years of age and older with eosinophilic e…
Tarpeyo
budesonide delayed-release
Calliditas Therapeutics
1 INDICATIONS AND USAGE TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are …
Elliotts B Solution
Buffered intrathecal electrolyte/dextrose injection
Lukare Medical, LLC
For the intrathecal administration of methotrexate and cytarabine for the prevention or treatment of meningeal leukemia and lymphocytic lymphoma.…
Suboxone
buprenorphine in combination with naloxone
Indivior, Inc.
1 INDICATIONS AND USAGE SUBOXONE sublingual film is indicated for treatment of opioid dependence. SUBOXONE sublingual film should be used as part of a…
Crysvita
burosumab-twza
Kyowa Kirin, Inc.
1 INDICATIONS AND USAGE CRYSVITA is a fibroblast growth factor 23 (FGF23) blocking antibody indicated for: The treatment of X-linked hypophosphatemia …
Busulfex
Busulfan
Otsuka Pharmaceutical Company, Ltd
1 INDICATIONS AND USAGE BUSULFEX is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic…
Berinert
C1 esterase inhibitor (human)
CSL Behring LLC
Treatment of acute attacks of hereditary angioedema.…
Ruconest
C1 ESTERASE INHIBITOR RECOMBINANT
Pharming Healthcare Inc.
1 INDICATIONS AND USAGE RUCONEST is a C1 esterase inhibitor [recombinant] indicated for the treatment of acute attacks in adult and adolescent patient…
Haegarda
C1 esterase inhibitor subcutaneous (human)
CSL Behring LLC
For routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. ||| HAEGARDA is a plasma-derived concentrate …
CABERGOLINE
CABERGOLINE
SOLA Pharmaceuticals, LLC
INDICATIONS AND USAGE Cabergoline Tablets, USP are indicated for the treatment of hyperprolactinemic disorders, either idiopathic or due to pituitary …
CABOMETYX
cabozantinib
Exelixis, Inc.
1 INDICATIONS AND USAGE CABOMETYX is a kinase inhibitor indicated for the treatment of patients with advanced renal cell carcinoma (RCC). ( 1.1 ) pati…
Cafcit
Caffeine
O.P.R. Development, L.P.
INDICATIONS AND USAGE CAFCIT (caffeine citrate) is indicated for the treatment of apnea of prematurity.…