Elevidys Clinical Development: Confidence In Surrogate Endpoint A Longstanding Concern For FDA Reviewers
Executive Summary
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.
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FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.
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CBER Director Peter Marks urged the review teams for Sarepta’s gene therapy to consider pre- and post-treatment videos showing improved physical function, but clinical and statistical reviewers cited limitations in the patient videos and said they failed to inform whether micro-dystrophin is a suitable surrogate endpoint for accelerated approval.
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