Regenerative Medicine Advanced Therapy Designation Launches

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In a FDAVoice blog post, CBER Director Peter Marks discusses the new designation for cell therapies that treat life-threatening diseases.

Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) announced in a FDAVoiceblog post, dated March 21, 2017, that the Regenerative Medicine Advanced Therapy (RMAT) Designation program had begun receiving designation requests from industry. The RMAT designation was created to help foster the development and expedite the approval of regenerative medicines that treat life-threatening diseases. Regenerative medicines include cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products.

Sponsors requesting RMAT designation can do so with or after submission of an investigational new drug application. The designation gives sponsors earlier interactions with FDA, and the application may receive priority review and accelerated approval. According to Marks, “meetings with sponsors of RMAT-designated products may include discussions of whether accelerated approval would be appropriate based on surrogate or intermediate endpoints reasonably likely to predict long-term clinical benefit, or reliance upon data obtained from a meaningful number of sites.”

FDA may permit fulfillment of post-approval requirements under accelerated approval once a product receives RMAT designation. This fulfillment would come through “the submission of clinical evidence, clinical studies, patient registries, or other sources of real-world evidence such as electronic health records; through the collection of larger confirmatory datasets; or through post-approval monitoring of all patients treated with the therapy prior to approval.”


“The FDA’s Center for Biologics Evaluation and Research is committed to helping make regenerative medicine advanced therapies that are shown to be safe and effective available as soon as possible, particularly for patients with serious or life-threatening diseases or conditions lacking other treatment options,” Marks stated in the blog post.

Source: FDA