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 it 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
The US regulator agreed to await the results of the Phase III EMBARK study before considering approval in a wider age group for the $3