A young patient being treated with the gene therapy had previously died from acute liver failure. It was found that this risk had not been previously reported in connection with Elevidy’s treatment.

The FDA’s Investigation

The FDA investigated the risk of acute liver failure and is evaluating the need for further regulatory action. The second patient was fifteen years old and enrolled in Sarepta’s Phase III ENVISION trial.

Sarepta’s CEO, Doug Ingram, said that liver injury is a known risk of AAV therapies and as far as he knew many  clinical-stage gene therapies use AAVs for their delivery system.

A BMO Capital Markets analyst commented in writing to his investors that the two deaths were “unsurprising”. He added that they were most likely due to the known AAV risk and known safety concerns associated with the use of these delivery systems.

Sarepta

Following the second Elevidys death Sarepta announced improved safety issues including a group of experts who will consider an “enhanced immunosuppression regimen”. Elevidys shipments will be temporarily halted by the company.

One possible improvement would be the addition of the drug “Sirolimus” to the regimen as it did not have an adverse effect on Elevidy’s function. Yet Sirolimus carries its own risks which is a potential increase of infections.

The company is using the Phase III pivotal study to seek approval of its accelerated consent to a full FDA approval.

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Rose Duesterwald      July 18, 2026