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.
Azithromycin Dihydrate
AZITHROMYCIN DIHYDRATE
PD-Rx Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Azithromycin is a macrolide antibacterial drug indicated for the treatment of patients with mild to moderate infections caused…
Cayston
aztreonam
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE CAYSTON ® is indicated to improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa . Safety a…
Lioresal Intrathecal
baclofen
Medtronic, Inc.
INDICATIONS AND USAGE Baclofen tablets, USP are useful for the alleviation of signs and symptoms of spasticity resulting from multiple sclerosis, part…
Colazal
Balsalazide disodium
Salix Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Balsalazide disodium capsules are indicated for the treatment of mildly to moderately active ulcerative colitis in patients 5 …
Simulect
Basiliximab
Novartis Pharmaceuticals Corporation
INDICATIONS AND USAGE Simulect ® (basiliximab) is indicated for the prophylaxis of acute organ rejection in patients receiving renal transplantation w…
SIRTURO
bedaquiline
Janssen Research & Development, LLC
1 INDICATIONS AND USAGE SIRTURO is a diarylquinoline antimycobacterial drug indicated as part of combination therapy in the treatment of adult and ped…
BLENREP
belantamab mafodotin-blmf
GlaxoSmithKline Intellectual Property Development Ltd.
1 INDICATIONS AND USAGE BLENREP is indicated in combination with bortezomib and dexamethasone for the treatment of adult patients with relapsed or ref…
Nulojix
belatacept
Bristol-Myers Squibb Company
1 INDICATIONS AND USAGE • NULOJIX is a selective T cell costimulation blocker indicated for prophylaxis of organ rejection in adult patients receiving…
Beleodaq
Belinostat
Acrotech Biopharma LLC
1 INDICATIONS AND USAGE Beleodaq is indicated for the treatment of adult patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This …
Rezurock
belumosudil
Kadmon Pharmaceuticals, LLC
1 INDICATIONS AND USAGE REZUROCK is indicated for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host dise…
Welireg
BELZUTIFAN
Merck Sharp & Dohme LLC
1 INDICATIONS AND USAGE WELIREG is a hypoxia-inducible factor inhibitor indicated: von Hippel-Lindau (VHL) disease for treatment of adult patients wit…
Bendeka
bendamustine for 50 ml admixture
Eagle Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE BENDEKA injection is an alkylating drug indicated for treatment of patients with: Chronic lymphocytic leukemia (CLL). Efficacy…
Treanda
Bendamustine hydrochloride
Cephalon, Inc.
1 INDICATIONS AND USAGE TREANDA is an alkylating drug indicated for treatment of patients with: Chronic lymphocytic leukemia (CLL). Efficacy relative …
Fasenra
benralizumab
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE FASENRA is an interleukin-5 receptor alpha-directed cytolytic monoclonal antibody (IgG1, kappa) indicated for: • add-on mainte…
Ammonul
benzoate/phenylacetate
Medicis Pharmaceutical Corp.
Adjunctive therapy in the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.…
Survanta intratracheal suspension
Beractant
Ross Laboratories
INDICATIONS AND USAGE SURVANTA is indicated for prevention and treatment (“rescue”) of Respiratory Distress Syndrome (RDS) (hyaline membrane disease) …
Orladeyo
berotralstat
BioCryst Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ORLADEYO ® is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 y…
Orladeyo
BEROTRALSTAT HYDROCHLORIDE
BioCryst Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ORLADEYO ® is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 y…
Cystadane
BETAINE
Recordati Rare Diseases
1 INDICATIONS AND USAGE CYSTADANE ® is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations in pediatr…
Betaine Anhydrous
BETAINE ANHYDROUS
Eton Pharmaceuticals, Inc.
1. INDICATIONS AND USAGE Betaine Anhydrous for Oral Solution is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood …
Betaloan Suik
BETAMETHASONE SODIUM PHOSPHATE AND BETAMETHASONE ACETATE
Advanced Rx Pharmacy of Tennessee, LLC
INDICATIONS AND USAGE When oral therapy is not feasible, the intramuscular use of Betamethasone Sodium Phosphate and Betamethasone Acetate Injectable …
Marbeta 25 Kit
BETAMETHASONE SODIUM PHOSPHATE AND BETAMETHASONE ACETATE, BUPIVACAINE HYDROCHLORIDE, POVIDINE IODINE
Asclemed USA, Inc.
1 INDICATIONS AND USAGE Bupivacaine Hydrochloride Injection is indicated in adults for the production of local or regional anesthesia or analgesia for…
Zynteglo
BETIBEGLOGENE AUTOTEMCEL
Genetix Biotherapeutics Inc.
1 INDICATIONS AND USAGE ZYNTEGLO is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell …
Avastin
bevacizumab
Genentech, Inc.
1 INDICATIONS AND USAGE Avastin is a vascular endothelial growth factor inhibitor indicated for the treatment of: Metastatic colorectal cancer, in com…
Targretin
Bexarotene
Eisai, Inc.
1 INDICATIONS AND USAGE TARGRETIN ® (bexarotene) Capsules are indicated for the treatment of cutaneous manifestations of cutaneous T-cell lymphoma in …
Normocarb HF
bicarbonate infusate
Dialysis Solutions, Inc.
Use as a replacement solution in Continuous Renal Replacement Therapy (CRRT) to replace water and to correct electrolytes and acid-base imbalances in …
Biktarvy
bictegravir, emtricitabine, and tenofovir alafenamide
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE BIKTARVY is indicated as a complete regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in adul…
Filsuvez
BIRCH TRITERPENES
Chiesi USA, Inc.
1 INDICATIONS AND USAGE FILSUVEZ is indicated for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in adul…
Blenoxane
Bleomycin sulfate
Bristol-Myers Squibb Pharmaceutical Research Institute
INDICATIONS AND USAGE Bleomycin for Injection, USP should be considered a palliative treatment. It has been shown to be useful in the management of th…