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.
- Hairy cell leukemia
- Hansen's disease
- Hemolytic uremic syndrome, atypical, childhood
- Hemophilia A, congenital
- Hemophilia B
- Heparin-induced thrombocytopenia
- Hepatic encephalopathy
- Hereditary angioedema
- Hereditary leiomyomatosis and renal cell cancer
- Herpes simiae (B virus)
- Herpes simplex encephalitis
- Herpes zoster oticus
- Herpesvirus simiae B virus
- Hodgkin lymphoma
- Homocysteinemia due to MTHFR deficiency
- Homocystinuria due to CBS deficiency
- Homocystinuria due to defect in methylation cbl e
- Homocystinuria due to defect in methylation cbl g
- Huntington disease
- Hurthle cell thyroid cancer
- Hypereosinophilic syndrome
- Hyperparathyroidism, neonatal severe primary
- Hyperparathyroidism, primary
- Hypogonadotropic hypogonadism without anosmia, X-linked
- Hypothalamic dysfunction
Click a disease on the left to see its medical product(s).