THE U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients after receiving Sarepta Therapeutics’ gene therapy, Elevidys.
In June, Sarepta reported a second death in a patient who had received its gene therapy, which raised concerns about the safety and future demand for the treatment. The patients who died were a 16-year-old, weighing 70 kilograms, and a 15-year-old, weighing 50 kilograms. Both boys were non-ambulatory and their deaths occurred within 90 days after treatment, the company said in an investor call last week.
The two patients showed signs of acute liver failure and were hospitalized less than two months after treatment with Elevidys, the health regulator said on Tuesday. It added that it is evaluating the need for further regulatory action.
Sarepta said in an email to Reuters, “The FDA communication was triggered by our report to the FDA and our suggested update to the label to include information relating to the recent events.”
The U.S. prescribing information for Elevidys warns of acute liver injury but does not mention liver failure or death.
Elevidys, approved by the FDA in 2024 for ambulatory Duchenne muscular dystrophy patients aged four and older, is the only gene therapy available for the disease. It carries a known risk of liver damage and was conditionally approved for non-ambulatory patients despite failing to meet the main goal in a late-stage study.
Sarepta said earlier this month it was investigating both cases independently and in relation to each other to identify any common risk factors.
The company has suspended its Elevidys sales forecast for 2025, halted shipments to non-ambulatory patients and is working with regulators on a new treatment plan.
