Following the death of a 16-year-old patient, Swiss pharmaceutical company Roche has suspended multiple clinical trials of the gene therapy Elevidys in Europe. The European Medicines Agency requested this temporary halt while investigators examine the circumstances surrounding the young patient’s death, which involved acute liver failure and a recent cytomegalovirus infection that may have played a contributing role.
The suspension affects three ongoing studies, including the critical global Phase 3 Envision trial, which was examining the therapy’s effectiveness in both ambulatory and non-ambulatory patients between ages eight and seventeen. This key study was anticipated to deliver results by 2027.
Elevidys, developed by Sarepta Therapeutics and licensed to Roche for markets outside the United States, has been administered to more than 800 patients to date. While liver injury is a known potential side effect of both Elevidys and similar gene therapies, this marks the first instance of a death related to acute liver failure among treated patients.
The news has significantly impacted Sarepta’s market performance, with shares declining more than 7% to approximately $58 in early trading. This continues a downward trend that began when the company first announced the patient death on March 18, when the stock was trading above $100.
Industry analyst Joseph Schwartz from Leerink Partners characterized the development as “another unfortunate development for a story that cannot seem to catch a break.” However, Schwartz and other analysts suggest that investors may be overreacting to the news, noting that the market response appears disproportionate to the situation.
The trial suspensions specifically affect new patient enrollment and dosing, while previously treated patients will continue to be monitored for data collection purposes. The impact on Sarepta’s revenue is expected to be relatively modest, as sales outside the U.S. represent a smaller portion of the company’s overall business.
In the United States, Elevidys currently holds approval for patients aged four and older with specific gene mutations. The therapy has received full approval for ambulatory patients within this group and accelerated approval for non-ambulatory patients, with the latter requiring confirmation through the now-suspended Envision study.
This development represents another challenge for Elevidys, which has achieved strong sales despite ongoing questions about its therapeutic benefits. The FDA’s initial approval in 2023 was limited to a specific subset of Duchenne patients, with an expansion of its clearance the following year, despite mixed clinical trial results in treating the muscle-wasting condition.
The temporary halt of these trials could potentially delay study outcomes and impact the ongoing regulatory review process in Europe. However, Roche emphasizes that the suspension is a precautionary measure while authorities complete their investigation into the patient death.
This situation highlights the complex nature of gene therapy development and the careful balance between advancing potentially life-changing treatments and ensuring patient safety. The temporary suspension of these trials represents a significant but necessary step in the thorough evaluation of serious adverse events in clinical research, particularly in the context of treating serious genetic conditions like Duchenne muscular dystrophy.