Patient Dies Following Treatment With DMD Gene Therapy Elevidys

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Elevidys is an adeno-associated virus vector-based gene therapy designed to deliver a shortened, functional component of dystrophin to skeletal muscle. The treatment received accelerated approval for ambulatory pediatric patients aged 4 to 5 years with DMD in 2023. Subsequently, the indication was expanded to include both ambulatory and nonambulatory DMD patients aged 4 years and older. 

Elevidys is administered as a single intravenous infusion. Prior to the infusion, health care providers are advised to evaluate the appropriateness of administering therapy. This assessment includes obtaining liver enzyme tests, as patients with preexisting liver impairment or acute liver disease (eg, acute hepatic viral infection) may be at increased risk for serious hepatic injury.

The patient in this case had suffered acute liver failure following the Elevidys infusion. While acute serious liver injury has been observed with Elevidys and is listed as a warning in the prescribing information, Sarepta noted in its safety update that “acute liver failure leading to death represents a severity of acute liver injury not previously reported for Elevidys, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.” 

It was also revealed that the patient had a recent cytomegalovirus infection, which may have contributed to the liver damage. In general, Elevidys therapy should be postponed in patients with infections.

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development. 

This article originally appeared on MPR