Sarepta falls after notifying of patient death following treatment with Elevidys

Shares of Sarepta (SRPT) Therapeutics are down 24.95, or 25%, to $76.40 in pre-market trading after the company shared a safety update related to Elevidys, which it notes is the “only approved gene therapy in patients with Duchenne muscular dystrophy.” The company stated: “We are profoundly saddened to share that a young man with Duchenne muscular dystrophy has passed away following treatment with Elevidys, having suffered acute liver failure. Acute liver injury is a known possible side effect of Elevidys and other AAV-mediated gene therapies and is highlighted in the prescribing information. Although it is not a new safety signal and the benefit-risk of Elevidys remains positive, 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. In addition, testing revealed this patient had a recent cytomegalovirus infection which was identified by the treating physician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis. Patient safety and well-being are Sarepta’s top priority. We continue to gather and analyze the information from this event. The event has been reported to the relevant health authorities and Sarepta intends to update the prescribing information to appropriately represent this event. We have also reported the event to Elevidys clinical study investigators and prescribing physicians.”