Sarepta reports Elevidys patient death, citing acute liver failure

Sarepta Therapeutics has reported a patient death following treatment with Elevidys, the only approved gene therapy for patients with Duchenne muscular dystrophy.

Following treatment with Elevidys, a young male patient suffered acute liver failure. While acute liver injury is a known possible side effect of Elevidys — as well as of other AAV-mediated gene therapies — acute liver failure leading to death represents a severity of acute liver injury not previously reported for Elevidys.

According to Sarepta, testing revealed that the patient had a recent cytomegalovirus infection, a common herpes virus infection, which could have been a contributing factor. The infection can damage the liver, resulting in CMV hepatitis.

Elevidys is a single-dose AAV-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne — mutations or changes in the DMD gene that result in the lack of dystrophin protein — through the delivery of a transgene that codes for the targeted production of Elevidys micro-dystrophin in skeletal muscle.

The FDA granted accelerated approval for Elevidys in June 2023 for ambulatory pediatric patients aged 4-5 years who have a confirmed mutation in the DMD gene. A year later, the agency approved a label expansion for Elevidys to include all individuals, regardless of ambulatory status, with DMD with a confirmed mutation in the DMD gene who are at least 4 years of age. 

To date, Elevidys has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.