FDA Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases

This guidance is intended for sponsors of drugs and biological products who are considering submitting requests for orphan-drug designation for their drugs under section 526 of the Federal Food, Drug, and Cosmetic Act (FD&C Act). The Food and Drug Administration (FDA) does not expect to grant any additional orphan-drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). Pediatric-subpopulation designations that have already been granted will not be affected by this change.