Orphan Drug
Definition
A drug developed to treat a rare disease or condition affecting fewer than 200,000 people in the United States. The Orphan Drug Act provides manufacturers with incentives including tax credits, grant funding, and 7 years of market exclusivity to encourage development.
Example
Ivacaftor (Kalydeco) was approved as an orphan drug for cystic fibrosis affecting fewer than 30,000 Americans.
Related Terms
Mentioned in Guides
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The Orphan Drug Act ExplainedWhy rare diseases were historically neglected by drug developers and how the 1983 Orphan Drug Act transformed treatment options for …
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Gene Therapy DrugsGene therapy aims to treat disease by modifying the genetic material inside a patient's cells. Learn how approved gene therapies …