Sarepta’s Elevidys will remain on the market with a black box warning. But this gene therapy for Duchenne muscular dystrophy still faces commercial headwinds and analysts say the company has work to do to win over patients and investors.
Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys. The company has proposed an immunosuppressive regimen to mitigate the liver injury risk posed by the therapy.
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Sarepta Therapeutics said the Duchenne muscular dystrophy patient recently had a cytomegalovirus infection, which can damage the liver and may have contributed to the fatality. It’s the first patient death out of more than 800 patients treated with Elevidys, a gene therapy for the rare muscle-wasting disease.
FDA reviewers concluded the data for Sarepta Therapeutics’ Elevidys were insufficient to show efficacy in the rare muscle disorder Duchenne muscular dystrophy. Peter Marks, the agency’s top biologics official, reviewed the same data and reached a different conclusion.
Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.