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Regenerative Medicine Advanced Therapy Designation

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

  1. The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
  2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  3. Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition

Based on FDA’s interpretation of Section 506(g) of the 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 FDA’s 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 Administration
Center for Biologics Evaluation and Research
Office of Tissues and Advanced Therapies
Document Control Center
10903 New Hampshire Avenue
WO71, G112
Silver 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.