Sarepta LGMD gene therapy trials placed on clinical hold by FDA

In a regulatory filing, the company announced that the U.S. Food and Drug Administration has placed a clinical hold on the company’s investigational gene therapy clinical trials for limb girdle muscular dystrophy. The hold includes the company’s LGMD clinical trials related to its product candidates SRP-9003, SRP-9004, SRP-6004, and SRP-9005, the filing noted. “The company previously announced on July 16, 2025 that it had paused each of the LGMD programs mentioned above as part of a strategic restructuring process, with the exception of SRP-9003. On December 18, 2024, the company announced completion of enrollment and dosing in SRP-9003-301, the Phase 3 clinical trial of SRP-9003. The company intends to seek to discuss with FDA the potential pathway to submit a Biologics License Application to FDA seeking accelerated approval for SRP-9003 after the clinical hold is lifted. Also on July 21, 2025, the company announced that FDA has revoked the platform technology designation for the company’s AAVrh74 platform technology previously granted on June 2, 2025,” the company stated.