Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label to include Duchenne Muscular Dystrophy Patients without Restriction to Age or Ambulatory Status

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) 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 (Study SRP-9001-301), 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 (Study SRP-9001-103), an open label clinical study in patients with Duchenne, that is enrolling patients ages 2 years and older. The supplement was submitted to the U.S. Food and Drug Administration (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 (PMR) and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval.

“Consistent with our patient commitment, Sarepta moved rapidly to submit an efficacy supplement to the biologics license application for ELEVIDYS seeking label expansion to remove age and ambulation restrictions from the approved indication,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “Understanding that Duchenne patients are in a race against time, we appreciate that the Agency also moved quickly to engage and provide us with guidance on the process for reviewing our BLA supplement.”

As part of a collaboration agreement signed in 2019, Sarepta Therapeutics is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Together, the companies are implementing a comprehensive joint clinical development plan to maximize the chances of broad approval and access so that ELEVIDYS can reach as many individuals with Duchenne as rapidly as possible.