Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged more than 27% in regular trading Tuesday following the company’s online statement about a patient death linked to its gene therapy product, ELEVIDYS. The incident involved acute liver failure in a patient treated with the therapy, sparking investor concerns and triggering a sharp sell-off.
Sarepta Therapeutics, a company that develops precision genetic medicines for rare diseases, expressed grief over the loss of a young man with Duchenne muscular dystrophy. The company acknowledged that acute liver injury is a known potential side effect of ELEVIDYS, as documented in the therapy’s prescribing information. To date, over 800 patients have received ELEVIDYS through clinical trials or prescriptions, but this marks the first reported case of fatal liver injury leading to death, casting a shadow over the therapy’s safety profile.
The physician treating the patient highlighted a recent cytomegalovirus (CMV) infection as a possible contributing factor to the liver failure. CMV hepatitis is known to cause liver damage and could have played a role in this case. Sarepta emphasized that patient safety is its top priority and said it is working diligently to collect and analyze information related to the incident.
The company has reported the event to relevant health authorities and plans to update ELEVIDYS prescribing information to include this case. It has also communicated the incident to clinical study investigators and physicians who prescribe the therapy.
The impact of this news on Sarepta Therapeutics (NASDAQ: SRPT) stock underscores how sensitive biotech companies are to safety concerns about their products. Investors often react quickly to any perceived risk linked to novel therapies, especially when patient safety is involved. As Sarepta navigates the aftermath of this event, the market’s reaction highlights the inherent challenges and responsibilities that genetic medicine developers must contend with.
