What safety problems emerged with Elevidys in 2025?
Three patients died from acute liver failure following treatment with Elevidys or a related Sarepta gene therapy using the same AAV vector, prompting the FDA to revoke the platform's technology designation and request a halt to all shipments.
Beginning in March 2025, patients died after treatment. CGTLive's timeline records a first death from acute liver failure in a non-ambulatory patient on March 18, 2025 and a second on June 15, after which dosing of non-ambulatory patients was suspended. A third death, in a 51-year-old man given a related Sarepta gene therapy (SRP-9004) for limb-girdle muscular dystrophy using the same AAVrh74 vector, also involved liver failure.
In response, the FDA placed Sarepta's limb-girdle gene therapy trials on hold, revoked the platform technology designation for the AAVrh74 vector, and asked Sarepta to stop shipping Elevidys. Sarepta also had to add a boxed warning for acute liver injury and failure.