Definition: An orphan drug is a medication or biologic, such as a vaccine or blood product, that is used to treat a rare disease or condition. A disease is considered by the U.S. Food and Drug Administration (FDA) to be rare if fewer than 200,000 people in the United States have it.
Examples:
The FDA has approved more than 250 orphan drugs providing treatment for more than 14 million people in the U.S. Examples include:
- Fabrazyme (agalsidase beta) used to treat fabry disease.
- Pumozyme (dornase alfa) used to treat cystic fibrosis.
