Sarepta announces filing of New Drug Application for Duchenne muscular dystrophy treatment

Sarepta Therapeutics Inc., a developer of RNA-targeted therapeutics, on Tuesday announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application for eteplirsen for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.

“We are pleased with the FDA’s acceptance of our NDA for eteplirsen, as it represents an important milestone, not only for Sarepta, but for the Duchenne community," Edward M. Kaye, interim CEO and chief medical officer, said. "We look forward to continuing to work closely with the FDA during the regulatory review process.”  

Approximately 13 percent of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by eteplirsen/exon 51 skipping.

“We believe eteplirsen has the potential to make a meaningful impact on the lives of patients amenable to skipping exon 51, and we aim to build on our experience with eteplirsen to work with the FDA to inform and potentially expedite the clinical and regulatory pathway for the follow on exons, with the goal of reaching as many patients amenable to exon skipping as possible,” Kaye said.

Sarepta Therapeutics is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases, such as drug-resistant bacteria and other rare human diseases.