Overview

• The FDA is probing two reports of acute liver failure deaths in non-ambulatory Duchenne muscular dystrophy patients treated with Elevidys.
• The patients were a 16-year-old weighing 154 pounds and a 15-year-old weighing 110 pounds, and both died within three months of treatment.
• Sarepta has halted commercial shipments of Elevidys for non-ambulatory individuals and paused dosing in its ENVISION trial for older and non-ambulatory patients.
• Elevidys, approved in 2024 for ambulatory patients aged 4 and older, has been used by over 900 patients despite earlier efficacy concerns.
• FDA regulators are evaluating whether to update safety warnings or take further actions, which could include market withdrawal of the gene therapy.