FDA Seeks Halt to Sarepta’s Duchenne Gene Therapy amid Third Patient Death

FDA asks Sarepta to halt Elevidys shipments after death in limb-girdle trial, spotlighting rare-disease gene therapy safety and trial oversight concerns.

The FDA asks Sarepta to stop shipping Elevidys, its Duchenne gene therapy, after rising safety flags. It marks 3rd death, the latest: a 51-year-old man in a Phase I limb girdle trial death, caused by gene therapy liver failure. The agency has called on Sarepta to cease distribution of Elevidys. But the company has pushed back, declining to halt shipments for ambulatory patients and insisting no new Elevidys safety alert exists for that group.

This is a pivotal point for gene therapy oversight. While Elevidys shipments to non-ambulatory Duchenne patients were paused, Sarepta stopped short of a full halt. The company attributes the limb girdle trial death to its investigational SRP-9004 program and says it notified regulators and clinicians. Still, doubts are rising. Sarepta, during a broad restructuring tied to layoffs and cuts, reportedly left out mention of the third patient death fueling questions around internal transparency. Striking the right balance between rapid accesses to life-saving treatments and ensuring patient safety is increasingly difficult. Should gene therapy liver failure gain recognition as a rising concern, the FDA could respond with tougher oversight. That shift might stall innovation, push back upcoming launches, and compel the industry to revisit clinical trial protocols across the board.

Externally, investors, regulators, and providers are watching closely. The Elevidys safety alert and recent limb girdle trial death raise the bar for what’s considered safe especially with liver risks tied to AAV vectors. If gene therapy liver failure continues to emerge, future programs could be squeezed. Deep investment is now needed in vector safety, tighter dose ranges, and post-approval tracking. The promise of rare-disease cures won’t hold if trust erodes. FDA asks Sarepta to stop shipping is no longer just a headline; it’s a signal to reset standards before another third patient death hits the system.