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.
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- Carbamoyl phosphate synthetase 1 deficiency
- Carcinoid syndrome
- Carcinoid tumor
- Carcinoid tumor childhood
- Cardiomyopathy due to anthracyclines
- Catecholaminergic polymorphic ventricular tachycardia
- CDK4 linked melanoma
- Cerebral palsy
- Chagas disease
- Char syndrome
- Childhood acute lymphoblastic leukemia
- Childhood hypophosphatasia
- Cholesteryl ester storage disease
- Chromophil renal cell carcinoma
- Chromophobe renal cell carcinoma
- Chronic graft versus host disease
- Chronic granulomatous disease
- Chronic inflammatory demyelinating polyneuropathy
- Chronic lymphocytic leukemia
- Chronic myeloid leukemia
- Chronic myeloproliferative disorders
- Citrullinemia type I
- Clear cell renal cell carcinoma
- Coccidioidomycosis
- Colorectal cancer, childhood
- Common variable immunodeficiency
- Congenital bile acid synthesis defect, type 1
- Congenital bile acid synthesis defect, type 2
- Congenital generalized lipodystrophy type 1
- Congenital generalized lipodystrophy type 2
- Congenital generalized lipodystrophy type 4
- Congenital herpes simplex
- Congenital sucrase-isomaltase deficiency
- Crohn's disease
- Cryopyrin-associated periodic syndrome
- Cryptococcosis
- Cryptosporidiosis
- Cushing's syndrome
- Cutaneous T-cell lymphoma
- Cystic fibrosis
- Cysticercosis
- Cystinosis
- Cystinuria
- Cytomegalovirus retinitis
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