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Sarepta’s External Controls Analysis Weakened By Elevidys Placebo Data Comparison

 
• By Sue Sutter

When placebo data from the only randomized trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s proffered external control data, the placebo subjects numerically outperformed the external controls, ‘calling into question the comparability of the external control group,’ an FDA statistical reviewer said.

Drug Review Profile: Elevidys
FDA reviewers did not see a case for Elevidys' efficacy in Sarepta's external controls comparison. • Source: Shutterstock

A US Food and Drug Administration statistician used placebo arm data from a Sarepta Therapeutics, Inc. clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) to highlight the challenges of trying to use external controls to demonstrate efficacy.

When placebo data from the only randomized, double-blind trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s proffered external control data, the placebo subjects numerically outperformed the external controls on a functional endpoint, “calling into

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