Issue Date: September 24, 2025
Issued by: Center for Biologics Evaluation and Research,Office of Therapeutic Products
Topic(s): Gene Therapy
Status: Draft
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions: Draft Guidance for Industry
This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions with our recommendations on the expedited development and review of these therapies, including as provided under section 506(g) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as added by section 3033 of the 21st Century Cures Act (Cures Act). Under section 506(g) of the FD&C Act, a regenerative medicine therapy can be designated as a regenerative advanced therapy if it meets certain criteria. FDA refers to such designation as “regenerative medicine advanced therapy” (RMAT) designation (see section III.C of this document). This guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as RMATs. To that end, the guidance provides information about the provisions in the Cures Act regarding the use of the accelerated approval pathway for regenerative medicine therapies that have been granted designation as an RMAT. Finally, the guidance describes considerations in the clinical development of regenerative medicine therapies and opportunities for sponsors of such products to interact with the Center for Biologics Evaluation and Research (CBER) review staff. As a general matter, this guidance addresses regenerative medicine therapies regulated by CBER as biological products under the FD&C Act, section 351 of the Public Health Service Act (PHS Act) (42 U.S.C. 262), and applicable regulations.
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