[2] RMAT goes beyond breakthrough therapy features by allowing for accelerated approval of drugs based on surrogate endpoints.
In order to qualify for RMAT status, a treatment must A regenerative medicine therapy is defined in section 506(g)(8) of the FD&C Act to include cell therapies, therapeutic tissue engineering, human cell and tissue products.
In this context, accelerated approval means approval based on The ability to use 'Real World Evidence' (RWE), i.e. post-market evidence of safety and effectiveness, is particularly useful in the context of orphan diseases, where recruiting a sufficiently large cohort for pre-marketing clinical trials may not be feasible.
Viralym-M[11] (Roctavian, Valrox) (Lentiglobin)[15] (Breyanzi) (Ologo)[16] Vertex Pharmaceuticals Transfusion-dependent beta thalassemia Castle Creek Biosciences St. Jude Children's Research Hospital In 2020, the FDA received 34 requests for RMAT status, of which 12 (35.3%) were granted.
RMAT designated drugs include the novel CAR-T therapy Kymriah and betibeglogene autotemcel for beta thalassemia.