FDA committee supports gene therapy for muscular dystrophy

DNA vector

The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy.

The vote supports the accelerated approval of Sarepta Therapeutics’ 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,” said Doug Ingram, President and Chief Executive Officer, Sarepta. “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.”

SRP-9001 is intended to treat the underlying cause of Duchenne, which is characterised by mutations in the dystrophin gene that results in the lack of dystrophin protein. In the absence of 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 application is supported by non-clinical evidence, in addition to efficacy and safety data from studies 101, 102 and 103, as well as an integrated analysis across these three clinical studies comparing functional results to a propensity-score-weighted external control.

The CTGTAC’s vote will be considered by the FDA when making its decision regarding the potential accelerated approval of SRP-9001. The Biologics License Application (BLA) for SRP-9001 is currently under priority review by the FDA.

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