StacksVerified U.S. regulatory reference

21 CFR §316.23

Verified against eCFR.gov as of June 20, 2026View official text on eCFR.gov
  1. (a)A sponsor may request orphan-drug designation at any time in its drug development process prior to the time that sponsor submits a marketing application for the drug for the same rare disease or condition.
  2. (b)A sponsor may request orphan-drug designation of an already approved drug for an unapproved use without regard to whether the prior marketing approval was for a rare disease or condition.