Sarepta Wins FDA Approval for Muscular Dystrophy Drug

Sarepta Therapeutics has received FDA approval of an expansion to the labeled indication for Elevidys, a single-dose, adeno-associated virus (AAV)-based gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD 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.

The approval expands the labeled indication for Elevidys to include the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age who are ambulatory and have a confirmed mutation in the DMD gene or who are non-ambulatory and have a confirmed mutation in the DMD gene.The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of Elevidys micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

“Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, president and chief executive officer, Sarepta.

As part of a collaboration agreement with Roche signed in 2019, 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. (Source: Sarepta Therapeutics Website, 20 June, 2024)