In a significant development for the neuromuscular disease community, an FDA advisory committee has given a positive recommendation for Sarepta Therapeutics’ gene therapy, Elevidys, targeting Duchenne muscular dystrophy (DMD). The committee’s vote comes after evaluating clinical trial data suggesting the therapy could alter the trajectory of this rare muscle-wasting disorder.
The advisory panel voted 8-4 in favor of the therapy for ambulatory children with DMD who are four years or older, citing evidence of increased dystrophin production—a protein essential for muscle strength. Although questions remain about the durability of the treatment’s benefit and which patient segments may benefit most, panel members agreed the unmet need and overall risk-benefit profile warranted support.
Sarepta CEO Doug Ingram called the decision “a pivotal step forward for Duchenne families,” emphasizing the years of collaboration between patients, advocacy groups, and clinical researchers. If granted full FDA approval, Elevidys would become the first gene therapy marketed for DMD in the United States.
The FDA is expected to make its final decision in the coming weeks. A green light would mark not only another milestone for Sarepta, but also a major leap forward for gene therapy in rare inherited diseases—potentially paving the way for a new era in neuromuscular care.
