Patient dies after using Sarepta's muscular dystrophy gene therapy

In a concerning development for the field of gene therapy, Sarepta Therapeutics, a company focused on innovative treatments for muscular dystrophy, reported the death of a young patient while participating in a clinical trial for its gene therapy. Occurring just days prior to the announcement on March 18, 2025, this incident has raised alarms about the safety and effectiveness of such treatments, particularly in the context of its expedited approval by the U.S. Food and Drug Administration (FDA) in 2023. The patient reportedly died from acute liver injury, which has been noted as a potential side effect of the therapy called Elevidys. Importantly, while acute liver injury is known, the severity of the case presented was unprecedented among over 800 patients who have undergone treatment since its introduction. The Elevidys gene therapy aims to provide a solution for Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration and weakness, mainly affecting males. The therapy uses a disabled virus to insert a replacement gene responsible for producing dystrophin, a protein crucial for muscle function. The costs associated with this cutting-edge therapy are significant, sitting at approximately $3.2 million for a one-time treatment, which places it out of reach for many families. Despite the high stakes involved, the FDA granted expedited approval, allowing for use in patients aged four and older, even those who cannot walk – a marked shift from previous restrictions. The demise of the patient has prompted Sarepta Therapeutics to consider updating the prescribing information for Elevidys to include warnings about the heightened risks associated with acute liver injury. In light of the recent death, the company is expected to conduct further assessments of the therapy, potentially reshaping its clinical direction and the way in which the medicine is marketed to healthcare professionals and patients. Investors reacted sharply to the news, with Sarepta's shares plummeting over 23% in morning trading, highlighting the uncertainty surrounding the company's future following this fatality. As gene therapy continues to evolve as a pioneering treatment modality for genetic disorders, the implications of patient safety and the incidence of severe side effects bring critical attention to the need for thorough clinical evaluations and transparent communication regarding risks. The outcome of ongoing studies, which are essential for securing full FDA approval for this therapy, has now become even more urgent, particularly given that none of the other therapies developed by Sarepta for DMD has been fully confirmed to work as of yet.