US FDA investigates third death after treatment with Sarepta's gene therapy Elevidys

 The FDA is intensifying its scrutiny of Sarepta Therapeutics following a third patient death linked to the company’s gene therapy programs. Just hours after Sarepta reported the death of a 51-year-old man with limb-girdle muscular dystrophy (LGMD) from acute liver failure (ALF) following SRP-9004 treatment, the agency ordered an immediate halt to all Elevidys shipments. While the therapies are different, both use the same viral vector, AAVrh74.

Earlier this year, two non-ambulatory teenagers also died from ALF after receiving Elevidys for Duchenne muscular dystrophy (DMD). In response, Sarepta voluntarily paused shipments of the therapy for non-ambulatory patients in June while it worked to develop a more robust immunosuppressive protocol. The company initially declined to extend this pause to ambulatory patients but has since reversed course and now says it will suspend all Elevidys shipments. Roche, which markets Elevidys outside the US, has also temporarily paused new orders in countries that reference the FDA for regulatory approval.

Alongside the shipping halt, the FDA paused all LGMD trials, requested a boxed warning for Elevidys, and rescinded AAVrh74’s platform designation. While analysts agreed the pause was likely the best decision given the circumstances, it’s unclear how the FDA’s investigation will play out or what the future of Sarepta will be. That uncertainty is felt most acutely by the Duchenne patient community, which is now left waiting to learn if, and when, Elevidys will become available again.

https://www.drugdiscoverynews.com/weekly-rundown-fda-probes-sarepta-after-third-death-in-gene-therapy-trial-16516