Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys has achieved better than expected initial sales, but its future hangs in the balance after its confirmatory Phase III study missed its primary endpoint.
The disappointing news that Elevidys (delandistrogene moxeparvovec-rokl) failed its Phase III EMBARK study was revealed on 30 October, creating uncertainty about whether the therapy would stay on the market in its current use in four and five year olds, see its
Key Takeaways
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Just days after the disappointing Phase III results for Elevidys, Sarepta has unveiled strong sales from its first three months on the market
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The gene therapy was granted accelerated approval in a restricted 4-5 year old group, but the US FDA must now decide how to respond to the primary endpoint failure
