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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- Malaria
- Malignant hyperthermia
- Malignant melanoma, childhood
- Malignant mesothelioma
- Mantle cell lymphoma
- Marginal zone lymphoma
- Mastocytosis
- Mastocytosis cutaneous with short stature conductive hearing loss and microtia
- Melanoma astrocytoma syndrome
- Melanoma, familial
- Merkel cell carcinoma
- Methylmalonic acidemia and homocysteinemia type cblX
- Methylmalonic acidemia with homocystinuria
- Methylmalonic acidemia with homocystinuria type cblC
- Methylmalonic acidemia with homocystinuria type cblD
- Methylmalonic acidemia with homocystinuria type cblF
- Methylmalonic acidemia with homocystinuria type cblJ
- Microscopic polyangiitis
- Mild phenylketonuria
- Muckle-Wells syndrome
- Mucopolysaccharidosis type I
- Mucopolysaccharidosis type II
- Mucopolysaccharidosis type IVA
- Mucopolysaccharidosis type VI
- Mucopolysaccharidosis type VII
- Multicentric Castleman Disease
- Multiple endocrine neoplasia type 2A
- Multiple myeloma
- Multiple sclerosis
- Myasthenia gravis
- Mycobacterium Avium Complex infections
- Mycosis fungoides
- Myelodysplastic syndromes
- Myelodysplastic/myeloproliferative disease
- Myelofibrosis
- Myopathic carnitine deficiency
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