List of FDA Orphan Drugs | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program

List of FDA Orphan Drugs | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program

List of FDA Orphan Drugs

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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.

T

• T-cell lymphoma 1A
• Testicular cancer
• Therapy related acute myeloid leukemia and myelodysplastic syndrome
• Thrombotic thrombocytopenic purpura, acquired
• Thyroid cancer, follicular
• Thyroid cancer, medullary
• Tièche-Jadassohn nevus
• Tourette syndrome
• Transverse myelitis
• Trichuriasis
• Trypanosomiasis, Human West-African
• Tuberculosis
• Tuberous sclerosis
• Tuberous sclerosis, type 1
• Tuberous sclerosis, type 2
• Tumor necrosis factor receptor-associated periodic syndrome
• Turner syndrome
• Tyrosinemia type 1