Sarepta And Roche Press Ahead With Elevidys Filing Despite Duchenne Trial Miss

Sarepta Therapeutics is to seek full approval for its Duchenne gene therapy Elevidys in the US and partners Roche have likewise vowed to seek approval in ex-US countries, despite the product failing to hit the primary endpoint of its Phase III study.

Elevidys (delandistrogene moxeparvovec-rokl) gained an accelerated US Food and Drug Administration approval in June to treat ambulatory patients ages four to five years with a confirmed mutation in the Duchenne muscular dystrophy gene, with an advisory committee having voted narrowly in favor of the move