Want An RMAT Designation? Please Include At Least Some Data, US FDA Tells Sponsors
CBER's Rachael Anatol says such a situation has happened "more than a few times"; instances appear to have been largely concentrated in earlier days of the RMAT program.
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Product characterization questions and partial or inconsistent clinical data are among the more common reasons why the US FDA denies, or seeks additional information about, a request for regenerative medicine advanced therapy designation, FDA’s Bryan says; sponsors should consider why they are seeking designation when deciding whether to apply for RMAT, breakthrough therapy or both.
Draft guidance suggests comparing therapies for a rare disease to each other and active control, offers examples of novel efficacy endpoints and what therapies may qualify for breakthrough and RMAT designations.
“It didn’t take long for folk to start applying,” a US Food and Drug Administration official says of the 17 applications the agency has received so far for designation under its new regenerative medicine advanced therapy (RMAT) program for fostering the development of such products.