Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a pioneer in precision genetic medicine for rare diseases, has announced today that the U.S. Food and Drug Administration (FDA) has accepted and filed the Company’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) (referred to as the “Efficacy Supplement”).
The primary objectives of the Efficacy Supplement are:
- To broaden the labeled indication for ELEVIDYS to include: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.”
- To transition ELEVIDYS from accelerated approval to traditional approval status.
The FDA has granted the Efficacy Supplement a Priority Review with a targeted review completion date of June 21, 2024. Additionally, the Agency has confirmed its decision not to convene an advisory committee meeting to discuss the supplement.
Doug Ingram, President, and Chief Executive Officer of Sarepta Therapeutics, expressed satisfaction with the FDA’s acceptance of the Efficacy Supplement, 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. We are particularly grateful for the Division’s prompt engagement and commitment to expediency by granting priority review and setting a June 21 review goal date. 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.”
Under a collaboration agreement established in 2019, Sarepta Therapeutics is collaborating with Roche to revolutionize the future for the Duchenne community, enabling individuals affected by the disease to maintain and safeguard their muscle function. Sarepta is responsible for securing regulatory approval and commercialization of ELEVIDYS in the U.S., as well as overseeing manufacturing. Roche, on the other hand, is accountable for securing regulatory approvals and ensuring ELEVIDYS reaches patients worldwide, excluding the U.S.
Patients and physicians seeking further information about ELEVIDYS can access it at www.SareptAssist.com or by calling 1-888-727-3782.
