FDA Finalizes Orphan Drug Rules, Updates Misinterpreted Terms

The FDA has finalized changes to orphan drug regulations, clarifying confusion around orphan drug designation and the terms that have hung up sponsors of rare-disease treatments. The new regulations, published in the June 11 Federal Register, finalize terms such as “orphan subset” and the criteria a drug must meet to be considered an orphan drug based on rule changes proposed in October 2011.
Washington Drug Letter