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.
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.”
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.
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.
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