Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug Administration (FDA) for accelerated approval for Duchenne muscular dystrophy (DMD).

The FDA committee voted 8 to 6 in favor of the investigational gene therapy for the treatment of ambulatory patients who have the rare, fatal neuromuscular genetic disease with a confirmed mutation in the DMD gene.

The positive vote of the FDA CTGTAC is driven by the assessment of the totality of the evidence, which includes the product design of the investigational gene therapy along with its biological and empirical data.

SRP-9001 is backed by non-clinical evidence apart from efficacy and safety data from the 101, 102, and 103 studies and an integrated analysis across the three clinical studies comparing the functional findings to a propensity-score-weighted external control (EC).

The recommendation from the CTGTAC is not binding but will be taken into consideration by the FDA when making its decision about granting the accelerated approval of SRP-9001.

Sarepta Therapeutics filed a biologics license application (BLA) with the FDA seeking accelerated approval for SRP-9001, which already has fast track designation, rare pediatric disease, and orphan drug status in the US. The FDA granted priority review to the gene therapy candidate with a regulatory action date of 29 May 2023.

Doug Ingram — Sarepta Therapeutics president and CEO said: “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.”

SRP-9001 is designed to treat the root cause of Duchenne muscular dystrophy, which is characterized by mutations in the dystrophin gene resulting in the absence of the dystrophin protein. If dystrophin which is essential to protect and strengthen muscles is not present, then muscles weaken and damaged.

According to Sarepta Therapeutics, the intention of SRP-9001 is to provide a gene that codes for a reduced, functional type of dystrophin to muscle cells.

  Doug Ingram, Duchenne muscular dystrophy, Muscular dystrophy, Sarepta Therapeutics, US Food and Drug Administration