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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.

Review documents show that in at least four separate meetings with Sarepta dating back to December 2018, Center for Biologics Evaluation and Research staff questioned whether a correlation

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