Sarepta’s Elevidys Under Scrutiny Following Fatal Liver Complication

A young patient diagnosed with Duchenne muscular dystrophy has passed away following the administration of Elevidys, the gene therapy developed by Sarepta Therapeutics. The company disclosed that the individual succumbed to acute liver failure, a known potential adverse effect associated with the therapy. While liver complications are a recognized risk of Elevidys and other gene therapies utilizing modified viral vectors, this marks the first reported fatality among the more than 800 patients treated either in clinical trials or commercially.
Sarepta has stated that laboratory tests identified a recent cytomegalovirus (CMV) infection in the patient. Although CMV often does not cause symptoms in healthy individuals, it can lead to severe complications in immunocompromised patients, including liver inflammation. A treating physician suggested that this viral infection may have been a contributing factor in the patient’s demise.
The biotechnology company has informed relevant regulatory authorities, clinical trial investigators, and medical professionals of the incident. Additionally, Sarepta has announced plans to revise Elevidys’ prescribing information to reflect the occurrence accurately. In a statement, the company reaffirmed its commitment to patient safety and stated that efforts are ongoing to gather and assess all available data related to the event.

The U.S. Food and Drug Administration (FDA) granted initial authorization for Elevidys in mid-2023 for a limited patient population, later expanding its approval to include a broader group. The therapy’s commercial performance has been notable, with reported sales reaching approximately $384 million in the last quarter, and some projections indicating potential annual revenues exceeding $2 billion.
However, the regulatory pathway for Elevidys was complex. The FDA’s decision to approve the treatment was met with internal debate, as some reviewers questioned the therapy’s efficacy. Approval was ultimately granted following intervention by Dr. Peter Marks, a key decision-maker within the agency, who highlighted supportive secondary study measures and historical data comparisons suggesting meaningful therapeutic effects.
Safety concerns surrounding gene therapies have been an ongoing topic of discussion within the medical community. The viral vectors used to deliver these treatments pose risks, including liver toxicity. Previous cases have been documented where gene therapy trials led to severe hepatic complications. For example, multiple fatalities occurred in clinical studies involving therapies from Audentes Therapeutics and Novartis, raising broader concerns about treatment-associated liver failure.
In response to such risks, medical experts have advocated for rigorous screening procedures, including assessments for preexisting liver conditions and limitations on dosing. Current FDA guidelines already recommend pre- and post-treatment corticosteroid administration for patients receiving Elevidys, along with routine liver enzyme monitoring over three months to detect potential hepatic complications.
At a recent medical conference, Sarepta presented safety data from 156 patients treated with Elevidys. Of these, eight cases of liver-related abnormalities were documented, but the company stated that no clinically significant cases were observed beyond 90 days post-treatment. Furthermore, all liver-related adverse events reported in the study were said to have resolved over time.

Parent Project Muscular Dystrophy, a patient advocacy organization, responded to the recent fatality by emphasizing the importance of further research to understand gene therapy-associated risks and ensure optimal patient safety. Physicians may now exhibit increased caution before prescribing the therapy, particularly given the ongoing debate regarding its clinical efficacy.
Sarepta’s Elevidys is currently the only approved gene therapy for the condition, but other companies are advancing their treatments. Regenxbio is conducting late-stage trials for its candidate, while Solid Biosciences has indicated its intention to engage with the FDA regarding potential accelerated approval for its experimental therapy.