List of FDA Orphan Drugs
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet.
A
- Acquired hemophilia A
- Acral lentiginous melanoma
- Acromegaly
- Acute intermittent porphyria
- Acute lymphoblastic leukemia
- Acute myeloid leukemia
- Acute promyelocytic leukemia
- Adenosine deaminase deficiency
- Adult-onset citrullinemia type II
- Alpha-1 antitrypsin deficiency
- Alpha-thalassemia
- Alveolar capillary dysplasia
- Amebiasis
- AML with myelodysplasia-related features
- Amyotrophic lateral sclerosis
- Amyotrophic lateral sclerosis type 10
- Amyotrophic lateral sclerosis type 11
- Amyotrophic lateral sclerosis type 2
- Amyotrophic lateral sclerosis type 3
- Amyotrophic lateral sclerosis type 4
- Amyotrophic lateral sclerosis type 5
- Amyotrophic lateral sclerosis type 6
- Amyotrophic lateral sclerosis type 7
- Amyotrophic lateral sclerosis type 8
- Amyotrophic lateral sclerosis type 9
- Amyotrophic lateral sclerosis-parkinsonism/dementia complex 1
- Anaplastic large cell lymphoma
- Anaplastic thyroid cancer
- Anterior uveitis
- Anthrax
- Aortic aneurysm, familial thoracic 4
- Aplastic anemia
- Arginase deficiency
- Argininosuccinic aciduria
- Aspergillosis
- Atypical hemolytic uremic syndrome
- Autosomal dominant polycystic kidney disease
- Autosomal dominant type B hypercholesterolemia
- Autosomal recessive juvenile Parkinson disease
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