By Fineline Cube 
The European Medicines Agency (EMA) may lift the temporary hold on certain Phase III studies for Elevidys (delandistrogene moxeparvovec), a Duchenne muscular dystrophy (DMD) gene therapy co-developed by Sarepta Therapeutics Inc. (NASDAQ: SRPT) and Roche (SWX: ROG, OTCMKTS: RHHBY). This decision follows the independent data monitoring committee (DMC) review, which concluded that the benefit-risk profile supports continuing the trials without protocol changes.
Adverse Event Review
The DMC met to evaluate the case of a DMD patient who died following treatment with Elevidys, due to acute liver failure. The committee’s assessment determined that the overall evidence supports maintaining the trials, citing a favorable benefit-risk profile.
Regulatory Response
Sarepta and Roche plan to submit the DMC’s findings to EU regulatory bodies within a week. The companies anticipate no significant delays in the studies affected by the temporary halt.
Disease Background
Duchenne muscular dystrophy (DMD) is a severe, X-linked degenerative neuromuscular disease caused by mutations in the dystrophin gene. Elevidys, an AAV-based gene therapy, delivers a transgene to promote micro-dystrophin production in skeletal muscle, addressing the genetic root of DMD.-Fineline Info & Tech