Sarepta submits efficacy supplement to expand Elevidys label

Sarepta announced submission of an efficacy supplement to the biologics license application, or BLA, for Elevidys to expand its labeled indication as follows “(ELEVIDYS is indicated for) the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.” The efficacy supplement is supported by results from EMBARK, a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study in patients with Duchenne between the ages of 4 through 7 years and data from ENDEAVOR, an open label clinical study in patients with Duchenne, that is enrolling patients ages two years and older. The supplement was submitted to the FDA with a request for Priority Review. An efficacy supplement is a submitted request for a proposed change to an approved product’s labeling, including adding or modifying an indication previously filed with the FDA. Sarepta has also completed the EMBARK postmarketing requirement and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval.