Sarepta Halts Duchenne Gene Therapy in Some Patients After Second Death

Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, to non-ambulatory patients following a second reported patient death due to acute liver failure2 5.

Both deaths occurred in non-ambulatory patients and were linked to the known risk of acute liver failure associated with adeno-associated virus (AAV)-based gene therapies like Elevidys5 3.

Dosing in the global Envision clinical trial, which evaluates Elevidys in older ambulatory and non-ambulatory individuals, has been paused with FDA agreement while the company evaluates a revised immunosuppression strategy2 3.

European regulators had already requested that Sarepta and Roche (the commercial partner) put temporary clinical holds on several Elevidys studies after the first fatality earlier in 20253 4.

More than 900 patients have received Elevidys through clinical trials and commercial use, but only about 140 non-ambulatory patients have been treated globally to date2 3.

Sarepta is assembling an independent panel of Duchenne and liver health experts to review a new immunosuppression approach to reduce risks2.

The recent news triggered a sharp decline in Sarepta's share price and caused concern and division in the Duchenne community over the risks and future use of the therapy1 5.