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.

N

• N-acetylglutamate synthase deficiency
• Narcolepsy
• Natal teeth, intestinal pseudoobstruction and patent ductus
• Neonatal Onset Multisystem Inflammatory disease
• Neonatal severe hyperparathyroidism
• Nephropathic cystinosis
• Neuroblastoma
• Neuronal ceroid lipofuscinosis 2
• Nodular melanoma
• Non 24 hour sleep wake disorder
• Noonan syndrome
• Noonan syndrome 1
• Noonan syndrome 2
• Noonan syndrome 3
• Noonan syndrome 4
• Noonan syndrome 5
• Noonan syndrome 6