MedCity News July 6, 2022
Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.
Gene therapy offers the potential to correct a genetic problem causing disease and Sarepta Therapeutics is among the companies vying to bring this treatment option to patients who have Duchenne muscular dystrophy. New clinical data indicate that Sarepta’s gene therapy led to improvement on a variety of measures of the muscle-wasting disorder. A regulatory submission is almost certain, but the question is when.
Sarepta has had ”very...
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