CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 16, 2024 07:00 AM Eastern Standard Time

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a pioneering force in precision genetic medicine for rare diseases, made a significant stride today as it announced the U.S. Food and Drug Administration (FDA) acceptance and filing of the company's efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) (the “Efficacy Supplement”).

The primary objectives of the Efficacy Supplement are two-fold: first, to broaden the labeled indication for ELEVIDYS to include the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene, and second, to transition the accelerated approval of ELEVIDYS to a traditional approval pathway.

Excitingly, the FDA has granted the Efficacy Supplement a Priority Review status, underscoring the agency's recognition of the potential impact of this treatment expansion. The review goal date has been set for June 21, 2024. Additionally, the FDA has confirmed that no advisory committee meeting is planned to discuss the supplement, signifying a streamlined regulatory process.

Doug Ingram, President and Chief Executive Officer of Sarepta Therapeutics, expressed his satisfaction with the FDA's decision, stating, “We are pleased to announce that FDA has accepted and filed Sarepta’s Efficacy Supplement to evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the approval from accelerated to traditional.”

Ingram further emphasized the company's commitment to expedited review, recognizing the urgency for families affected by Duchenne muscular dystrophy. He added, “Understanding that every day matters to families living with Duchenne, we will work with our regulatory counterparts to successfully complete this review as rapidly as possible."

Sarepta's collaboration with Roche, initiated in 2019, aims to transform the landscape for individuals affected by Duchenne muscular dystrophy, safeguarding their muscle function and enhancing their quality of life. Under this agreement, Sarepta holds responsibility for regulatory approval and commercialization of ELEVIDYS in the U.S., along with its manufacturing operations, while Roche is tasked with regulatory approvals and global distribution beyond the U.S.

This milestone represents a pivotal moment in the journey toward expanding treatment options for Duchenne muscular dystrophy and providing hope to countless individuals and families affected by this devastating disease.