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

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...