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.
Prolastin
Alpha1-proteinase inhibitor (human)
Bayer Corporation
For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emp…
Hexalen
Altretamine
Medimmune Oncology, Inc.
Single agent palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy with a cisplatin and/or alkylat…
Gocovri
Amantadine hydrochloride
Adamas Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE GOCOVRI ® is indicated: For the treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy,…
Ambrisentan
AMBRISENTAN
Cipla USA Inc.
1 INDICATIONS AND USAGE Ambrisentan tablets are indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) in adult patients: …
Firdapse
AMIFAMPRIDINE PHOSPHATE
Catalyst Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE FIRDAPSE ® is indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years…
Ethyol
Amifostine
Medimmune Oncology, Inc.
1 INDICATIONS AND USAGE ETHYOL is a cytoprotective agent indicated for: – reduction of cumulative renal toxicity associated with repeated administrati…
Aminocaproic Acid
AMINOCAPROIC ACID
American Regent, Inc.
INDICATIONS AND USAGE Aminocaproic Acid Injection is useful in enhancing hemostasis when fibrinolysis contributes to bleeding. In life-threatening sit…
Cordarone
Amiodarone HCl
Wyeth-Ayerst Laboratories
For initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in p…
Ammonium Lactate
AMMONIUM LACTATE
REMEDYREPACK INC.
INDICATIONS AND USAGE Ammonium Lactate Cream, 12% is indicated for the treatment of ichthyosis vulgaris and xerosis.…
Abelcet
Amphotericin B lipid complex
Liposome Company, Inc.
Treatment of invasive fungal infections in patients who are refractory to or intolerant of conventional amphotericin B therapy.…
NexoBrid
anacaulase-bcdb
Vericel Corporation
1 INDICATIONS AND USAGE NEXOBRID is indicated for eschar removal in adults and pediatric patients with deep partial thickness (DPT) and/or full thickn…
Agrylin
Anagrelide
Takeda Development Center Americas, Inc.
1 INDICATIONS AND USAGE AGRYLIN is indicated for the treatment of patients with thrombocythemia, secondary to myeloproliferative neoplasms, to reduce …
Kineret
anakinra
Swedish Orphan Biovitrum AB (publ)
1 INDICATIONS AND USAGE KINERET is an interleukin-1 receptor antagonist indicated for: Rheumatoid Arthritis (RA) Reduction in signs and symptoms and s…
EBANGA�
ansuvimab-zykl
Emergent Manufacturing Operations Baltimore LLC
treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including neonates born to a mother who is RT-PCR positive for Zair…
BioThrax
Anthrax Vaccine Adsorbed
Emergent BioDefense Operations Lansing LLC
For post-exposure prophylaxis of disease following suspected or confirmed Bacillus anthracis exposure, when administered in conjunction with recommend…
Cyfendus
Anthrax Vaccine Adsorbed, Adjuvanted
Emergent Product Development Gaithersburg, Inc.
post-exposure prophylaxis of disease following suspected or confirmed exposure to Bacillus anthracis in persons 18 through 65 years of age when admini…
FEIBA
ANTI-INHIBITOR COAGULANT COMPLEX
Takeda Pharmaceuticals America, Inc.
1 INDICATIONS AND USAGE FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for: Control and…
Alphanate
Antihemophilic factor (human)
Grifols Biologicals Inc.
For surgical and/or invasive procedures in patients with von Willebrand Disease (VWD) in whom desmopressin is either ineffective or contraindicated. I…
Kogenate
Antihemophilic factor (recombinant)
Bayer Corporation
1 INDICATIONS AND USAGE ALTUVIIIO is indicated for use in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency) for: Rou…
Eloctate
antihemophilic factor (recombinant), Fc fusion protein
Bioverativ Therapeutics, Inc.
Indicated in adults and children with Hemophilia A (congenital Factor VIII deficiency) for on-demand treatment and control of bleeding episodes, perio…
ALTUVIIIO
ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC-VWF-XTEN FUSION PROTEIN-EHTL
Bioverativ Therapeutics Inc.
1 INDICATIONS AND USAGE ALTUVIIIO is indicated for use in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency) for: Rou…
OBIZUR
antihemophilic factor (recombinant), porcine sequence
Takeda Development Center Americas, Inc.
Treatment of bleeding episodes in adults with acquired hemophilia A.…
Humate-P
Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized
CSL Behring
(1) In adult patients for treatment and prevention of bleeding in hemophilia A (classic hemophilia) and (2) in adult and pediatric patients for treatm…
CroFab
Antivenin, crotalidae polyvalent immune Fab (ovine)
Protherics, Inc.
Treatment of minimal and moderate North American Crotalidae envenomation…
Adzynma
APADAMTASE ALFA
TAKEDA PHARMACEUTICALS AMERICA, INC.
1 INDICATIONS AND USAGE ADZYNMA (ADAMTS13, recombinant-krhn) is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13…
Apokyn
Apomorphine HCl
MDD US Operations, LLC
For the acute, intermittent treatment of hypomobility, "off" episodes ("end-of-dose-wearing-off" and unpredictable :on/off" episodes) associated with …
Onapgo
apomorphine hydrochloride
MDD US Operations, LLC
1 INDICATIONS AND USAGE ONAPGO is indicated for the treatment of motor fluctuations in adults with advanced Parkinson's disease. ONAPGO is a dopaminer…
APRACLONIDINE
APRACLONIDINE
Sandoz Inc
INDICATIONS AND USAGE Apraclonidine ophthalmic solution 0.5% is indicated for short-term adjunctive therapy, in patients on maximally tolerated medica…