Sarepta Submits Biologics License Application for Duchenne Treatment

 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche.

The BLA is submitted for accelerated approval based on the expression of SRP-9001 dystrophin protein, an internally shortened and functional version of dystrophin, as a surrogate endpoint reasonably likely to predict clinical benefit. Among other things, the BLA is based on positive pre-clinical, biomarker and clinical functional results. In clinical trials, SRP-9001 demonstrated positive results at multiple time points, including one-, two- and four-years after treatment, in addition to a consistent safety profile. The submitted BLA for SRP-9001 includes efficacy and safety data from Studies SRP-9001-101, SRP-9001-102, SRP-9001-103 (also known as ENDEAVOR), as well as an integrated analysis across these three clinical studies comparing functional results to a propensity-score-matched external control (EC). Quantification of the SRP-9001-protein expression is measured by western blot and supported by immunofluorescence and efficacy is further supported by biomarker and clinical functional benefit as measured by the North Star Ambulatory Assessment (NSAA) and secondary timed tests.

Sarepta has proposed its fully-enrolled study EMBARK (Study SRP-9001-301) as the post-marketing confirmatory study to support the accelerated approval. EMBARK is a global, randomized, double-blind, placebo-controlled clinical trial. The primary endpoint for EMBARK is the assessment of the change in NSAA total score from baseline to week 52 compared to placebo.

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