Sarepta Therapeutics has announced that the U.S. Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.
“Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies. With the May 29 action date our top priority, we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001. We extend our sincere appreciation to the families, clinicians, FDA presenters and committee members who participated in today’s panel and to all those who provided input and comments both in the written record and in the open public hearing.”Doug Ingram, President and Chief Executive Officer, Sarepta
SRP-9001 is intended to treat the underlying cause of Duchenne, which is characterized by mutations in the dystrophin gene that results in the lack of dystrophin protein. Without dystrophin, which is required to strengthen and protect muscles, muscles become weakened and damaged. SRP-9001 is intended to deliver a gene that codes for a shortened, functional form of dystrophin to muscle cells. The committee’s positive vote is based on the evaluation of the totality of the evidence, including the SRP-9001 product design as well as biological and empirical data. SRP-9001 is supported by non-clinical evidence in addition to efficacy and safety data from studies 101, 102 and 103 and an integrated analysis across these three clinical studies comparing functional results to a propensity-score-weighted external control (EC).
While not binding, the CTGTAC’s vote will be considered by the FDA when deciding on the potential accelerated approval of SRP-9001. The Biologics License Application (BLA) for SRP-9001 is currently under priority review by the FDA, with a regulatory action date of May 29, 2023.