Sarepta Submits BLA For SRP-9001 To Treat Ambulant Patients With Duchenne Muscular Dystrophy

(RTTNews) - Genetic medicine maker Sarepta Therapeutics, Inc. (SRPT) announced Thursday 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.

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