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GUIDANCE DOCUMENT

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions Guidance for Industry February 2019

Final Level 1 Guidance

This guidance is being distributed for comment purposes only.

Docket Number:
FDA-2017-D-6159
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions1 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).2 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 CBER review staff.


Submit Comments

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2017-D-6159.

Questions?

Contact Point
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
ocod@fda.hhs.gov
(800) 835-4709
(240) 402-8010