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

CAMBRIDGE, Mass. —  Sarepta Therapeutics, Inc., 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.

 

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV) based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the dystrophin gene that result in the lack of dystrophin protein – through the delivery of a shortened transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is a one-time infusion indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene and is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ELEVIDYS has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

In addition to EMBARK, which serves as the postmarketing confirmatory study, ELEVIDYS has been evaluated in three clinical studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Accelerated approval was primarily based on data from SRP-9001-102 and SRP-9001-103.

 

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.