Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy treatment.
The patient was a young man who suffered acute liver failure after being treated with Elevidys, according to a March 18 company news release. Acute liver injury is a known possible side effect of the drug. The patient had also received cytomegalovirus injections, which can also infect and damage the liver, and could have been a contributing factor in his death.
Sarepta said this is the first known death in more than 800 patients who have been treated with the drug in clinical trials or that were prescribed therapy.
“Patient safety and well-being are Sarepta’s top priority,” the company said. “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.”