Sarepta Pauses Duchenne Gene Therapy Trial After Second Patient Death

Sarepta Therapeutics has temporarily halted its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, after a second patient died from acute liver failure following treatment. The company announced it would pause a clinical trial and stop shipping the therapy to certain patients as it evaluates the safety protocols. Both patients who died were non-ambulatory, raising significant concerns about the therapy’s risks, especially for those with advanced stages of the disease.

Elevidys, priced at $3.2 million per patient, was approved by the FDA under its accelerated approval pathway, designed to fast-track treatments for life-threatening diseases. The therapy aims to address the genetic cause of Duchenne, which leads to progressive muscle weakness. However, the deaths have prompted a rethinking of the risk-benefit balance for families who were already weighing the therapy against the disease's devastating progression.

Sarepta’s stock dropped more than 40% following the disclosure of the second death. Patient advocacy groups have expressed concern, with CureDuchenne's Michael Kelly noting that the deaths have significantly altered the decision-making process for families. The risks associated with Elevidys now seem much more pronounced, leaving many families feeling uncertain about whether the potential benefits outweigh the dangers.

The FDA is closely monitoring the situation and is treating the issue with "the highest level of concern." Sarepta has pledged to investigate the cause of the liver failure and is consulting with an independent expert group to explore additional protocols, such as immune suppression, to prevent future liver-related complications. This situation highlights the growing debate over accelerated drug approvals, with some critics questioning whether the FDA’s flexibility with limited evidence is putting patients at risk.

Despite these challenges, Sarepta remains committed to addressing the safety concerns and continuing its clinical trials. The company is working closely with the FDA to determine the best path forward. As the reviews continue, the future of Elevidys and similar gene therapies will depend on the ongoing evaluation of their long-term safety and effectiveness.

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Created: June 16th, 2025

Citations:

Gilbert, D. (2025, June 16). Biotech pauses trial after second patient death linked to gene therapy. The Washington Post. https://www.washingtonpost.com/business/2025/06/16/sarepta-elevidys-duchenne-patient-death/