Skip to Main Content

In an unexpected move, the Food and Drug Administration will continue to apply exclusive marketing rights for so-called orphan drugs under its existing regulations, rather than take a broader approach suggested by a federal court in a highly controversial case involving one such medicine.

Going forward, the agency will continue to grant seven years of marketing exclusivity for specific uses or indications for orphan drugs — the regulatory term for rare disease medicines — instead of allowing a drug company to win more expansive marketing rights, even if the medicine is only approved for some uses for treating a particular disease.


The FDA decision, which was announced in a Federal Register notice on Monday, was made in response to a September 2021 ruling by a U.S. appeals court. That ruling found the agency acted “arbitrarily and capriciously” when it approved a particular orphan drug, because the approval contradicted federal law governing such designations.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!


Create a display name to comment

This name will appear with your comment

There was an error saving your display name. Please check and try again.