Commercial Fate Of Sarepta’s DMD Gene Therapy Remains Tied To EMBARK

Sarepta Therapeutics, Inc. expects the US Food and Drug Administration to grant an accelerated approval for its gene therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) – but initially only for children 4-5 years old and with an action date that the agency delayed slightly, to 22 June.

The company updated investors on 24 May on the regulatory outlook for the closely watched gene therapy, which is poised to be the first gene therapy to treat boys with the progressively debilitating and eventually fatal genetic disease