Top FDA official Peter Marks overruled staff to approve Sarepta gene therapy
What was the outcome of Elevidys' Phase 3 trial?
Elevidys' Phase 3 trial failed to meet its primary objective, showing a non-significant increase compared to placebo on the North Star Ambulatory Assessment (NSAA) scale, which measures motor function in patients aged four to seven with Duchenne muscular dystrophy2. However, the FDA considered the totality of the evidence, including secondary endpoints and the urgent unmet medical need, and expanded the approval of Elevidys.
What drug did the FDA expand approval for?
The FDA expanded approval for Elevidys, a gene therapy developed by Sarepta Therapeutics, for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory and non-ambulatory individuals aged 4 and older with a confirmed mutation in the DMD gene.
How many times has Sarepta influenced FDA decisions?
Sarepta Therapeutics has influenced FDA decisions three times, convincing top officials to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy3. The most recent decision expanded the approval of their gene therapy, Elevidys, to cover nearly all patients, regardless of age or wheelchair status, despite the drug failing a large Phase 3 trial last year5.