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FDA Regenerative Medicine Advanced Therapy (RMAT) Designation is now live

March 29, 2017

Healthcare Worker in a Doctor's Office.

FDA Regenerative Medicine Advanced Therapy (RMAT) Designation is now live.

Regenerative medicine encompasses a wide scope of innovative products including:

  • cell therapies e.g., chimeric antigen receptor T-cells (CAR-T cells) and human tissues grown on scaffolds
  • therapeutic tissue engineering products
  • human cell and tissue products
  • certain combination products using such therapies

Recognizing the importance of Regenerative Medicine, Congress included several provisions related to regenerative medicine field in the 21st Century Cures Act, signed into law on Dec. 13, 2016. Building on the FDA’s existing expedited programs available to regenerative medicine products, one of these provisions established a new program to help foster the development and approval of these products: Regenerative Medicine Advanced Therapy (RMAT) Designation.

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

  • 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;
  • The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  • Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition

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

FDA will take action on the requests within 60 calendar days of receipt.