Rare Diseases with FDA-Approved Medical Products
The Food and Drug Administration has charged the Office of Orphan Products Development (OOPD) to dedicate its mission to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD interacts with the medical and research communities, professional organizations, academia, governmental agencies, and the pharmaceutical industry, as well as rare disease groups.
The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful--more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.
The FDA has approved one or more orphan products for the treatment of the diseases listed below.
- Malignant melanoma, childhood
- Malignant mesothelioma
- Mantle cell lymphoma
- Mastocytosis cutaneous with short stature conductive hearing loss and microtia
- Melanoma astrocytoma syndrome
- Melanoma, familial
- Methylmalonic acidemia with homocystinuria
- Methylmalonic aciduria with homocystinuria cbl f
- Methylmalonicacidemia with homocystinuria cbl d
- Microscopic polyangiitis
- Morquio syndrome A
- Muckle-Wells syndrome
- Mucopolysaccharidosis type I
- Mucopolysaccharidosis type II
- Mucopolysaccharidosis type VI
- Multiple endocrine neoplasia type 2A
- Multiple myeloma
- Multiple sclerosis
- Multiple system atrophy (MSA) with orthostatic hypotension
- Mycosis fungoides
- Myelodysplastic syndromes
- Myelodysplastic/myeloproliferative disease
- Myopathic carnitine deficiency
Click a disease on the left to see its medical product(s).