RMAT Designation from the 21st Century Cures Act now live
The 21st Century Cures Act, signed into law on December 13, 2016, includes several provisions related to regenerative medicine. Regenerative medicine covers a wide range of innovative products including cell therapies, therapeutic tissue engineering products, human cell and tissue products, and certain combination products using such therapies. Examples of regenerative medicine include chimeric antigen receptor T-cell (CAR-T cell) treatments (FDA recently granted IND approval to the first gene-edited CAR-T cell therapy in the US), human tissues grown on scaffolds for subsequent use, and more.
The Regenerative Medicine Advanced Therapy (RMAT) Designation is a product of the Cures Act. The RMAT designation builds on FDA’s existing expedited programs available to regenerative medicine products and was established to foster the development and approval of these products. In an FDA blog, Peter Marks, M.D., Ph.D. (director of the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration), explains that “sponsors of certain products may obtain RMAT designation for their drug product if the drug is intended to tread serious or life-threatening diseases or conditions and if there is preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for that disease or condition.”1 The RMAT designation applies to:
- Certain cell therapies
- Therapeutic tissue engineering products
- Human cell and tissue products
- Certain combination products
Marks goes on to explain the process and benefits of the RMAT designation, which is summarized below:
- Sponsors may make such a request with or after submission of an investigational new drug application and the agency then will take action on the requests within 60 calendar days of receipt
- Sponsors of RMAT-designated products are eligible for increased and earlier interactions with the FDA
- Sponsors may be eligible for priority review and accelerated approval
- Once approved, the FDA can permit fulfillment of post-approval requirements under accelerated approval through the submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, when appropriate
The FDA has already begun receiving RMAT designation requests and they will likely receive more as regenerative medicine research and treatments continues to advance. Our experts work diligently with our clients to accelerate the product development process. We have helped countless sponsors receive priority review and expedited approval for their product, such as with the Breakthrough Therapy Designation. Contact us today to discuss how we can help with your next investigational new drug application.