The Swiss pharmaceutical company Roche has suspended multiple clinical trials of the gene therapy Elevidys in Europe following the death of a 16-year-old patient with Duchenne muscular dystrophy. The temporary halt came at the request of the European Medicines Agency pending a complete investigation into the cause of death.
The patient experienced acute liver failure and had recently contracted a cytomegalovirus infection, which may have played a role in the fatal outcome. The therapy’s developer, Sarepta Therapeutics, which licensed European rights to Roche, emphasized that this was the first death from acute liver failure among more than 800 patients who have received Elevidys treatment, though liver complications are a known risk associated with this and similar gene therapies.
Three clinical trials have been affected by the suspension, including the crucial global Phase 3 Envision study examining the therapy’s effects in both ambulatory and non-ambulatory patients between 8 and 17 years old. While new patient enrollment and dosing have been paused, those who have already received the one-time treatment will continue to be monitored for data collection purposes.
The development represents another challenge for Elevidys, which has achieved strong commercial performance despite ongoing questions about its efficacy. The U.S. Food and Drug Administration initially approved the therapy in 2023 for a limited group of Duchenne patients, later expanding its authorization despite mixed clinical trial results.
The news of the patient death in March triggered a significant decline in Sarepta’s stock value, which continued to fall following President Trump’s tariff announcement. The company’s shares dropped more than 7% to approximately $58 in early trading, marking a substantial decrease from over $100 before the March 18 death announcement.
Industry analysts, including Leerink Partners’ Joseph Schwartz, suggest that investors may be overreacting to the setback. While acknowledging the reputational damage to Elevidys, some believe the market response has been excessive. The impact on Sarepta’s revenue is expected to be relatively modest, as sales outside the United States represent a smaller portion of the company’s business.
In the U.S. market, Elevidys maintains its approval for patients aged four and older with specific gene mutations. The therapy has received full approval for ambulatory patients within this group, while holding accelerated approval for non-ambulatory patients. The latter authorization requires confirmation through the now-suspended Envision study, which was previously expected to yield results in 2027.
The research suspension adds uncertainty to the ongoing European regulatory review process. Roche’s communication to the patient community indicated that the clinical holds are temporary measures while authorities complete their investigation into the patient death. The incident highlights the delicate balance between advancing potentially transformative treatments and ensuring patient safety in gene therapy development.
The therapy’s journey has been marked by both promise and challenges, reflecting the complex nature of developing treatments for severe genetic disorders. While Elevidys has demonstrated commercial success and received expanded regulatory clearance in the U.S., this latest setback underscores the ongoing scrutiny faced by novel gene therapies and the importance of rigorous safety monitoring in clinical development programs.
The suspension of these trials comes at a critical time for both Roche and Sarepta, as they work to establish Elevidys as a treatment option for Duchenne muscular dystrophy patients globally. The outcome of the investigation into the patient death and subsequent regulatory decisions could have significant implications for the therapy’s future development and availability in European markets.