Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades

The death this week of a teenager receiving Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy is a tragic reminder of the stakes involved in cutting-edge biotech innovation.

While gene therapies like Sarepta’s offer an opportunity to treat and even cure diseases, that benefit carries risks. And although Elevidys has been used by more than 800 patients according to Sarepta, the recent death reflects concerns experts had with the therapy prior to its accelerated FDA approval in 2023 and full nod last year.

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Despite a failed late-stage trial, Sarepta pointed to the treatment’s effect on secondary evidence of a functional mechanism of action. And Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, ultimately overruled regulatory staff and a review team when he expanded the therapy’s label to treat a wider range of patients.

The 16-year-old boy whose death was reported this week suffered from acute liver failure. While liver injury has been listed as a possible side effect of Elevydis and other gene therapies, no previous reaction was this severe...