The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
Pictured: Sarepta sign on a brick building/courtesy Sarepta Therapeutics
Sarepta Therapeutics announced Friday the FDA has accepted and filed its efficacy supplement to the Biologics License Application for the company’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl).
The efficacy supplement is seeking to expand the labeled indication for Elevidys to treat patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. The supplement also looks to convert the Elevidys accelerated approval to a traditional approval.
The FDA has given the supplement a priority review and a target decision date of June 21, 2024. The agency will also not hold an advisory committee meeting for the supplement.
Sarepta’s stock price increased over 12% in premarket trading on Friday morning.
“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,” Sarepta CEO Doug Ingram said in a statement. “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.”
Elevidys accelerated approval was granted by the FDA in June 2023 for use in ambulatory pediatric DMD patients between the ages of four and five years with a confirmed mutation in the DMD gene. The treatment targets the root genetic cause of the disease by delivering a gene that codes a shortened form of dystrophin to muscle cells.
However, a confirmatory trial in October 2023 showed that Elevidys fell short of its primary efficacy endpoint and could not significantly improve functional mobility versus placebo. However, at the time, Sarepta still touted the results as evidence of clinical benefit.
The company is still forging ahead with its DMD treatments. In late January, Sarepta presented positive data from a Phase II trial of its candidate SRP-5051 (Vesleteplirsen) for treating patients with DMD amenable to exon 51 skipping. While the results did show some positive statistical results, some serious adverse events were recorded.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
