Top FDA official Peter Marks overrules staff decision to approve Sarepta gene therapy

For the third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to override the popular view of its staff and approve a drug for Duchenne muscular dystrophy.

On Thursday evening, the FDA announced it had expanded its approval of Alevidis, Sarepta’s Duchenne gene therapy, to include nearly all patients in its scope, regardless of their age or wheelchair status, despite the fact that the drug failed a large Phase 3 trial last year.

Documents released by the agency alongside the ruling show that the decision was made almost exclusively by Peter Marks, director of the agency's Center for Biologics Evaluation and Research. He overruled the judgment of three review teams and two top officials who wrote that the data submitted by Sarepta “create significant uncertainty about the benefits of the treatment.”