Second patient dies after gene therapy raises safety concerns

In the United States, Sarepta Therapeutics has faced significant backlash following the report of a second death associated with its gene therapy product, Elevidys. The biotech company revealed on June 16, 2025, that a 15-year-old boy died after receiving the treatment. This follows an earlier incident involving a 16-year-old boy who also succumbed to complications known as acute liver injury, recognized as a serious side effect of the gene therapy. Both patients had been treated for Duchenne muscular dystrophy (DMD), a severe genetic disorder that primarily affects males and leads to muscle degeneration and loss of mobility. Elevidys is the first gene therapy approved by the Food and Drug Administration (FDA) aimed at treating a specific genetic variant of DMD and has been available since its expedited approval in 2023. Following the recent deaths, Sarepta announced that it would pause shipments of Elevidys for older, non-ambulatory patients, as they receive larger doses of the therapy, potentially increasing risk factors. The company indicated it would assemble an expert panel to establish new safety protocols for the drug's administration, including the possibility of increased use of immune-system suppressing medications. This decision aims to address concerns among regulators, particularly the FDA, as the agency has shown reluctance about approving the drug due to potential ineffectiveness and safety issues. The drop in Sarepta's stock value highlights the market's apprehension regarding regulatory actions that could impact the future availability of Elevidys, leading to speculation that the FDA might impose further restrictions or necessary reviews. This upheaval raises questions about the efficacy of accelerated drug approvals and emphasizes the need for comprehensive safety evaluations to ensure patient well-being. Without these precautions, company executives fear that they may not only lose treatment options for patients but they will also face dire consequences for the future of their product on the market. Clearly, the repercussions of these incidents are profound, affecting not just the company’s stock performance, but also the lives of many families dependent on effective treatments for muscular dystrophy.