Gene Therapy Gets Priority Review for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) seeking accelerated approval for delandistrogene moxeparvovec (SRP-9001) for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD).

Delandistrogene moxeparvovec is an investigational gene transfer therapy designed to deliver a shortened, functional component of dystrophin to muscle tissue. The BLA is supported by efficacy and safety data from the SRP-9001-101, SRP-9001-102, and SRP-9001-103 studies (ClinicalTrials.gov Identifier: NCT03375164, NCT03769116, NCT04626674, respectively), along with an integrated analysis across these 3 studies that compared functional results to a propensity-score-matched external control.

Positive results were observed in more than 80 treated patients across multiple studies and multiple time points, including 1-, 2-, and 4-years after treatment, with a consistent safety profile. Under the accelerated approval program, the FDA can approve a treatment earlier based on a surrogate endpoint thought to predict clinical benefit, which in this case would be the expression of SRP-9001 dystrophin protein.

The safety and efficacy of the gene therapy is also being investigated in 125 patients 4 to 7 years of age with DMD in a global, randomized, double-blind, placebo-controlled phase 3 trial (EMBARK; ClinicalTrials.gov Identifier: NCT05096221). The EMBARK study is expected to serve as the post-marketing confirmatory trial.

A regulatory action date of May 29, 2023 has been set for the application.

This article originally appeared on MPR