Overview

• The FDA lifted its week-long pause on Elevidys deliveries to ambulatory Duchenne patients after finding a recent 8-year-old fatal liver event was unrelated to the therapy.
• Sarepta’s shares jumped more than 16% in after-hours trading, reflecting investor relief over the drug’s near-term sales outlook.
• Non-ambulatory patients remain under voluntary hold as the FDA requires a safety label update and additional clinical data before resuming treatment.
• Elevidys became the first U.S. gene therapy for Duchenne muscular dystrophy with accelerated approval in 2023 and was expanded last year to older patients who can no longer walk.
• FDA officials have indicated that new safety study results will be necessary before lifting restrictions on non-ambulatory patient use.