Overview

• Sarepta halted shipments of Elevidys for non-ambulatory Duchenne muscular dystrophy patients after reporting a second death from acute liver failure.
• Both fatalities involved non-ambulatory patients who suffered acute liver failure, highlighting elevated risks in those who have lost ambulation.
• The ENVISION trial evaluating Elevidys in older ambulatory and non-ambulatory patients has been paused pending safety revisions.
• Sarepta is working with external experts to develop an enhanced immunosuppressive regimen aimed at reducing liver toxicity, subject to FDA approval.
• Shares of Sarepta tumbled nearly 40% in premarket trading following the shipment halt and renewed safety concerns.