Regenerative Medicine Advanced Therapy Designation | FDA

As described in Section 3033 of the 21st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:

Based on FDAs interpretation of Section 506(g) of the Federal Food, Drug, and Cosmetic Act (as added by Section 3033 of the 21st Century Cures Act), certain human gene therapies and xenogeneic cell products may also meet the definition of a regenerative medicine therapy. For more information on FDAs definition of regenerative medicine therapies, refer to the Guidance for Industry, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.

The request for RMAT designation must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND. We will not grant a RMAT designation if an IND is on hold or is placed on hold during the designation review.

You may submit a request for RMAT designation to:

Food and Drug AdministrationCenter for Biologics Evaluation and ResearchOffice of Tissues and Advanced TherapiesDocument Control Center10903 New Hampshire AvenueWO71, G112Silver Spring, MD 20993-0002

If the RMAT designation request is submitted to your IND as an amendment, the cover letter should specify that the submission contains a REQUEST FOR REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION in bold, uppercase letters. If the request is submitted with an initial IND, the cover letter should specify that the submission contains both an INITIAL INVESTIGATIONAL NEW DRUG SUBMISSION and REQUEST FOR REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION in bold, upper case letters.

No later than 60 calendar days after receipt of the designation request, the Office of Tissues and Advanced Therapies (OTAT) will notify the sponsor as to whether RMAT designation has been granted. If OTAT determines that the RMAT designation request was incomplete or that the drug development program does not meet the criteria for RMAT designation, OTAT will include a written description of the rationale for such determination.

10/06/2021

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Regenerative Medicine Advanced Therapy Designation | FDA