Overview

• The FDA resumed shipments of Elevidys to ambulatory Duchenne patients after concluding that an eight-year-old’s death was unrelated to the therapy.
• Elevidys treatments for non-ambulatory patients remain on voluntary hold while Sarepta and the FDA negotiate additional safety measures and label updates.
• Sarepta has agreed to FDA requests for a black-box warning on acute liver failure and will work on risk-mitigation strategies for broader patient use.
• Shares of Sarepta Therapeutics surged more than 30% in premarket trading on news that U.S. availability had been restored.
• Elevidys, the first U.S. gene therapy for Duchenne muscular dystrophy, received accelerated approval in 2023 and carries a list price around $3.2 million per patient.