Studies supporting label expansion already are under way, with more targeting those currently ineligible, including boys with pre-existing antibodies, set to begin later this year, after the initial accelerated approval only allowed use in four- and five-year old patients.
EMBARK data could be sent to the FDA before the application to expand Elevidys label is submitted. • Source: Shutterstock
Sarepta Therapeutics, Inc.’s ongoing EMBARK trial to confirm the clinical benefit of its Duchenne muscular dystrophy gene therapy Elevidys also will serve as the foundation for a substantial label expansion expected in 2024.
Elevidys (delandistrogene moxeparvovec-rokl) received an accelerated approval from the US Food and Drug Administration on 22 June for four- and...
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