Press release
Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
- The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\n- The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators\n\n\n- Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place\n\n\n- ENVISION study is paused while seeking a protocol amendment to incorporate additional immunosuppression\n\n\n- Sarepta to host investor call on June 16, 2025, at 8:00 am Eastern time\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams.\n\n\nKey Safety Initiatives\n\n\nEvaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel's recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance.\n\n\nSuspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place.\n\n\nFor ambulatory patients, no treatment changes are being ...