Sarepta Pauses Gene Therapy Shipments Following Patient Deaths

Sarepta Therapeutics halted shipments of its moxeparvovec-rokl (Elevidys) gene therapy following a request from the Food and Drug Administration. FDA’s action came in response to three patient deaths from acute liver failure associated with Sarepta’s AAVrh74 adeno-associated virus vector-based gene therapies. Two cases involved non-ambulatory pediatric patients with Duchenne muscular dystrophy (DMD), while a third occurred in an adult patient with limb girdle muscular dystrophy who was enrolled in a clinical trial.

In addition, FDA placed clinical holds on Sarepta’s gene therapy trials for limb girdle muscular dystrophy and revoked the company’s platform technology designation, which was issued last month, due to these safety concerns.