In the next few weeks, Sarepta Therapeutics will release new data that could change the future of the company and the lives of thousands of patients with Duchenne muscular dystrophy.
The Phase 3 trial is testing whether Sarepta’s gene therapy, Elevidys, can slow progression of the fatal, muscle-wasting disease significantly more than placebo. The one-time treatment was given accelerated approval — a provisional authorization, pending confirmatory data — in June for 4- and 5-year-olds, despite concerns from reviewers about its actual benefit.
The forthcoming data could determine whether Elevidys remains available for those children and whether the Food and Drug Administration expands eligibility to include older children whose families have been desperately waiting. The data could also clarify the exact benefit those families can expect.