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Elevidys Clinical Development: Confidence In Surrogate Endpoint A Longstanding Concern For FDA Reviewers

 
• By Sue Sutter

Timeline of the gene therapy’s clinical development and regulatory review shows agency staff repeatedly questioned the ability of micro-dystrophin to predict clinical benefit in Duchenne muscular dystrophy patients.

Drug Review Profile: Elevidys
• Source: Shutterstock

Throughout Sarepta Therapeutics, Inc.’s clinical development of the gene therapy Elevidys (delandistrogene moxeparvovec-rokl, formerly SRP-9001), US Food and Drug Administration review staff repeatedly questioned the ability of micro-dystrophin, a surrogate endpoint, to predict clinical benefit in Duchenne muscular dystrophy patients.

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