Press release
Sarepta Therapeutics Provides Update on ELEVIDYS
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":" CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.\n\nFollowing the safety update on acute liver failure that was issued on March 18, European Union (EU) reference member country authorities requested that the independent data monitoring committee (DMC) meet to review the adverse event. While the analysis is being finalized, recruitment and dosing in certain clinical studies of ELEVIDYS are temporarily halted.\n\nThe independent DMC met on April 3 and concurred that based on the totality of evidence, the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols. At the request of EU regulators, Sarepta and Roche will submit this information in a response to the temporary halt within a week. Evaluation of the submission and the subsequent decision for lifting the temporary halt will follow the EU regulatory process.\n\nThe clinical studies affected by the temporary halt are Study SRP-9001-302 (ENVOL), Study SRP-9001-303 (ENVISION) and Study SRP-9001-104. Monitoring and data collection for already-enrolled participants continues, and we do not anticipate a material impact on the timeline for these studies.\n\nAbout ELEVIDYS (delandistrogene moxeparvovec-rokl)\nELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.\n\nELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.\n\n\nFor patients who are ambulatory and have a confirmed mutation in the DMD gene\n\n\nFor patients who are non-ambulatory and have a confirmed mutation in the DMD gene.\n\n\nThe DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin...