CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 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) (the “Efficacy Supplement”).
The goals of the Efficacy Supplement are twofold:
- To expand the labeled indication for ELEVIDYS as follows: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.”
- To convert the ELEVIDYS accelerated approval to a traditional approval.
The FDA has granted the Efficacy Supplement a Priority Review with a review goal date of June 21, 2024. The Agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.
“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,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “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.”
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.
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 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.
ELEVIDYS has been evaluated in four clinical studies: SRP-9001-101, SRP-9001-102, SRP-9001-103 (ENDEAVOR) and SRP-9001-301 (EMBARK). Accelerated approval of ELEVIDYS was primarily based on data from SRP-9001-102 and SRP-9001-103. The EMBARK study serves as the postmarketing confirmatory trial.
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.
Contacts
Investor:
Ian Estepan, 617-274-4052
[email protected]
Media:
Tracy Sorrentino, 617-301-8566
[email protected]