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.
Renacidin Irrigation
Citric acid, glucono-delta-lactone and magnesium carbonate
United-Guardian, Inc.
Leustatin Injection
Cladribine
R. W. Johnson Pharmaceutical Research Institute
1 INDICATIONS AND USAGE Cladribine tablets are indicated for the treatment of relapsing form of multiple sclerosis (MS), to include relapsing-remittin…
CLINDAMYCIN PHOSPHATE
CLINDAMYCIN PHOSPHATE
Rising Pharma Holdings, Inc.
INDICATIONS AND USAGE Clindamycin phosphate topical lotion is indicated in the treatment of acne vulgaris. In view of the potential for diarrhea, bloo…
Onfi
clobazam
Lundbeck, Inc.
1 INDICATIONS AND USAGE ONFI ® (clobazam) is indicated for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patie…
Clolar
clofarabine
Genzyme Corporation
1 INDICATIONS AND USAGE Clofarabine injection is indicated for the treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute…
Duraclon
Clonidine
Roxane Laboratories, Inc.
INDICATIONS AND USAGE Duraclon is indicated in combination with opiates for the treatment of severe pain in cancer patients that is not adequately rel…
Clotic
clotrimazole
Laboratorios Salvat, S.A.
1 INDICATIONS AND USAGE CLOTIC is indicated for the treatment of fungal otitis externa (otomycosis) due to Aspergillus species and Candida species in …
Mononine
Coagulation factor IX
Armour Pharmaceutical Company
1 INDICATIONS AND USAGE REBINYN, Coagulation Factor IX (Recombinant), GlycoPEGylated, is a recombinant DNA-derived coagulation Factor IX concentrate i…
Ixinity
COAGULATION FACTOR IX (RECOMBINANT)
Medexus Pharma, Inc.
1 INDICATIONS AND USAGE IXINITY, Coagulation Factor IX (Recombinant), is a human blood coagulation factor indicated in adults and children with hemoph…
ALPROLIX
Coagulation factor IX (recombinant), Fc fusion protein
Bioverativ Therapeutics, Inc.
Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or…
REBINYN
COAGULATION FACTOR IX RECOMBINANT, GLYCOPEGYLATED
Novo Nordisk
1 INDICATIONS AND USAGE REBINYN, Coagulation Factor IX (Recombinant), GlycoPEGylated, is a recombinant DNA-derived coagulation Factor IX concentrate i…
NovoSeven
Coagulation factor VIIa (recombinant)
Novo Nordisk, Inc.
1 INDICATIONS AND USAGE SEVENFACT is indicated for the treatment and control of bleeding episodes occurring in adults and adolescents 12 years of age …
Sevenfact
COAGULATION FACTOR VIIA RECOMBINANT HUMAN
Laboratoire Franais du Fractionnement et des Biotechnologies Socit Anonyme (LFB S.A.)
1 INDICATIONS AND USAGE SEVENFACT is indicated for the treatment and control of bleeding episodes occurring in adults and adolescents 12 years of age …
COAGADEX
Coagulation factor X (human)
Bio Products Laboratory Limited
1 INDICATIONS AND USAGE COAGADEX, Coagulation Factor X (Human), is a plasma-derived human blood coagulation Factor indicated in adults and children wi…
ANDEXXA
Coagulation factor Xa (recombinant), inactivated-zhzo
Alexion Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ANDEXXA is indicated for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life…
TRETTEN
coagulation factor XIII A-subunit (recombinant)
Novo Nordisk, Inc.
Routine prophylaxis of bleeding in patients with congenital Factor XIII A-subunit deficiency.…
Tybost
cobicistat
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE TYBOST is a CYP3A inhibitor indicated to increase systemic exposure of atazanavir or darunavir (once daily dosing regimen) in …
Cotellic
cobimetinib
Genentech, Inc.
1 INDICATIONS AND USAGE COTELLIC ® is a kinase inhibitor indicated: For the treatment of adult patients with unresectable or metastatic melanoma with …
Colcrys
colchicine
AR Holding Company, Inc.
1 INDICATIONS AND USAGE COLCRYS (colchicine, USP) is an alkaloid indicated for: Prophylaxis and treatment of gout flares in adults ( 1.1 ). Familial M…
Exosurf Neonatal for Intratracheal Suspension
Colfosceril palmitate, cetyl alcohol, tyloxapol
Glaxo Wellcome Inc.
Prevention of hyaline membrane disease (HMD), also known as respiratory distress syndrome (RDS), in infants born at 32 weeks gestation or less and in …
XIAFLEX
collagenase clostridium histolyticum
Auxilium Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE XIAFLEX is indicated for the treatment of adult patients with Dupuytren’s contracture with a palpable cord. XIAFLEX is indicat…
Alhemo
CONCIZUMAB
Novo Nordisk
1 INDICATIONS AND USAGE Alhemo is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric pat…
ALIQOPA
copanlisib
Bayer US LLC
Treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies…
Detectnet
copper Cu 64 dotatate
RadioMedix Inc.
1 INDICATIONS AND USAGE Detectnet is indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neur…
Zycubo
copper histidinate
Sentynl Therapeutics, Inc.
1 INDICATIONS AND USAGE ZYCUBO is indicated for the treatment of Menkes disease in pediatric patients. ZYCUBO is a copper replacement product indicate…
Acthrel
Corticorelin ovine triflutate
Ferring Laboratories, Inc.
To differentiate between pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushing's syndrome.…
Crenessity
crinecerfont
Neurocrine Biosciences, Inc.
1 INDICATIONS AND USAGE CRENESSITY is indicated as adjunctive treatment to glucocorticoid replacement to control androgens in adults and pediatric pat…