Orphan drug

orphan drug

n.

Any of various drugs or biologicals that may be useful in treating disease but are not considered to be commercially viable.

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Orphan drug

A term for a drug that treats a rare disease, defined by the Food and Drug Administration (FDA) as one that affects fewer than 200,000 Americans. The FDA has an Office of Orphan Products Development (OOPD), which offers grants to researchers to develop these products.

Mentioned in: Down Syndrome, Huntington Disease, Polycythemia Vera, Severe Combined Immunodeficiency