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.

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• Barrett esophagus
• B-cell lymphoma
• Benign essential blepharospasm
• Beta-thalassemia
• Bidirectional tachycardia
• Bile acid synthesis defect, congenital, 4
• Bladder cancer
• Blastic plasmacytoid dendritic cell
• Botulism
• BRCA1 hereditary breast and ovarian cancer syndrome
• BRCA2 hereditary breast and ovarian cancer syndrome