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.

H

• Hairy cell leukemia
• Hansen's disease
• Hemangioma
• Hemolytic uremic syndrome, atypical, childhood
• Hemophagocytic lymphohistiocytosis
• Hemophagocytic lymphohistiocytosis, familial, 2
• Hemophagocytic lymphohistiocytosis, familial, 3
• Hemophagocytic lymphohistiocytosis, familial, 4
• Hemophilia A
• Hemophilia B
• Heparin-induced thrombocytopenia
• Hepatic encephalopathy
• Hereditary angioedema
• Hereditary antithrombin deficiency
• Hereditary antithrombin deficiency type 2
• Hereditary antithrombin deficiency type I
• Hereditary diffuse gastric cancer
• Hereditary leiomyomatosis and renal cell cancer
• Hereditary orotic aciduria without megaloblastic anaemia
• Hereditary paraganglioma-pheochromocytoma
• Hereditary renal cell carcinoma
• Herpes simiae (B virus)
• Herpes simplex encephalitis
• Herpes zoster oticus
• Herpesvirus simiae B virus
• Hidradenitis suppurativa
• HIV/AIDS
• Hodgkin lymphoma
• Hodgkin lymphoma, childhood
• Homocystinuria
• Homocystinuria due to CBS deficiency
• Homocystinuria due to defect in methylation cbl e
• Homocystinuria due to defect in methylation cbl g
• Homocystinuria due to MTHFR deficiency
• Huntington disease
• Hurthle cell thyroid cancer
• Hydatidosis
• Hypereosinophilic syndrome
• Hyper-IgD syndrome
• Hyperkalemic periodic paralysis
• Hypokalemic periodic paralysis
• Hypophosphatasia
• Hypothalamic dysfunction