Rare Disease Drug Approvals

The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. The FDA grants the Orphan Drug designation (ODD) to products that could be used to treat a rare disease. While drugs receiving the ODD can qualify for special incentives, they are still subject to the typical FDA review process.

This report highlights 24 rare disease drug approvals from 2022, including three gene therapies. These products span eight therapeutic areas, with the most common being oncology.