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Sarepta Plots Multi-Study Label Expansion For Elevidys To Eventually Cover Nearly All DMD Population

 
• By Derrick Gingery

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.

Dart and gene therapy
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 five-year-old ambulatory DMD patients with a confirmed mutation of the...

Key Takeaways

  • Eventually, Sarepta expects 95% of DMD patients to be eligible for Elevidys

  • An sBLA asking for the label expansion for older...

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