Sarepta Therapeutics experienced a significant drop in stock value on Monday, falling nearly 50% after the announcement of a second patient death linked to its Elevidys drug, which is intended for treating Duchenne muscular dystrophy. This patient succumbed to acute liver failure (ALF), mirroring the fate of another patient reported in March.
Both individuals who died were non-ambulatory and undergoing treatment for Duchenne muscular dystrophy. In response to these tragic events, Sarepta has temporarily halted shipments of Elevidys for non-ambulatory patients and plans to ‘immediately convene an independent group of leading experts in Duchenne and liver health’ to review and potentially enhance the immunosuppression regimen associated with the drug.
The company is also putting on hold a clinical study assessing the efficacy of Elevidys for older patients, both ambulatory and non-ambulatory.
Louise Rodino-Klapac, Sarepta’s chief science officer and head of research and development, stated that the firm is taking ‘immediate, decisive steps to better understand and mitigate the risk of acute liver failure’ for patients receiving Elevidys.
This reaction from investors follows a prior decline in Sarepta’s stock in March when the first ALF-related death was announced. At that time, the company highlighted that acute liver injury was a recognized potential side effect and indicated plans to update the prescribing information accordingly based on the new findings.
As a result of these developments, Sarepta’s stock has plummeted to its lowest value in nine years.