A patient with Duchenne muscular dystrophy has died following treatment with Elevidys, a gene therapy developed by Sarepta Therapeutics, the company announced in a safety update. The news sent the company’s stock down about 23 percent in morning trading on March 18.
“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,” a company report read.
“Patient safety and well-being are Sarepta’s top priority. We continue to gather and analyze the information from this event,” the company added.
Sarepta Therapeutics, a major player in genetic medicine, stated that while acute liver injury is a known risk associated with Elevidys and other “AAV-mediated gene therapies,” acute liver failure leading to death had not previously been reported among the more than 800 patients treated with the therapy….