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Sarepta Therapeutics' Bid to Expand DMD Gene Therapy Indication Under FDA Priority Review

NEW YORK – The US Food and Drug Administration has accepted supplementary efficacy data as part of a biologics license application submitted by Sarepta Therapeutics, which is seeking to expand the indication for its Duchenne muscular dystrophy gene therapy.

Cambridge, Massachusetts-based Sarepta on Friday said the FDA has accepted new data as part of its BLA that it filed with the agency in December in the hopes of expanding the indication for Elevidys (delandistrogene moxeparvovec-rokl) to patients of all ages and converting its accelerated approval to full approval. In June 2023, the FDA granted accelerated approval to the gene therapy for patients with Duchenne muscular dystrophy who are 4 or 5 years old and have a confirmed mutation in the DMD gene that causes the progressive muscle disease, even though the company had sought an indication in all ages in its original BLA.

The FDA has granted Sarepta's efficacy supplement priority review and expects to issue a decision by June 21. The agency said it is not planning to hold an advisory committee meeting to discuss the supplement, according to Sarepta.

Elevidys is an adeno-associated virus vector-based gene therapy that delivers a copy of a gene that encodes for microdystrophin, a protein engineered by Sarepta. Microdystrophin is designed to carry out the normal functions of dystrophin, which is deficient in patients with Duchenne muscular dystrophy.

Elevidys is being developed and commercialized under a 2019 deal between Sarepta and Roche, in which Sarepta is responsible for regulatory approval and commercialization in the US, while Roche is responsible for those activities in other regions globally.