Following the death of a 16-year-old patient with Duchenne muscular dystrophy, Swiss pharmaceutical company Roche has suspended enrollment and treatment in three European clinical trials of the gene therapy Elevidys. The European Medicines Agency requested this temporary pause while investigators examine the circumstances surrounding the patient’s death.
The deceased patient experienced acute liver failure and had a recent cytomegalovirus infection that may have played a role in the outcome. According to Sarepta Therapeutics, which developed Elevidys and licensed it to Roche for markets outside the United States, this represents the first fatal case of acute liver failure among more than 800 patients who have received the treatment, though liver
complications are a recognized potential side effect of both Elevidys and similar gene therapies.
This development marks another challenge for Elevidys, which has achieved significant commercial success despite ongoing questions about its efficacy. The therapy initially received FDA approval in 2023 for a limited group of Duchenne patients, followed by an expanded authorization the subsequent year, despite mixed clinical trial results.
The announcement of the patient death in March triggered a significant decline in Sarepta’s stock value, which continued to fall amid broader market reactions to President Trump’s tariff announcement. The company’s shares dropped over 7% to approximately $58 in early trading, marking a substantial decrease from their pre-announcement level of above $100.
Industry analyst Joseph Schwartz from Leerink Partners characterized the situation as “another unfortunate development for a story that cannot seem to catch a break.” However, Schwartz and other analysts suggest that market reaction has been overly negative, arguing that investors are excessively discounting Sarepta’s value.
While the clinical holds could impact study timelines and the ongoing European regulatory review, analysts note that sales outside the U.S. represent a relatively minor portion of Sarepta’s revenue potential. Importantly, the suspension only affects new patient enrollment and dosing; patients who have already received the one-time treatment will continue to be monitored for data collection purposes, according to Roche.
Among the affected studies is the Envision trial, a crucial global Phase 3 study examining the therapy’s effects in non-ambulatory patients and ambulatory patients between ages eight and 17. Results from this trial had been anticipated in 2027.
Currently in the United States, Elevidys is approved 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 Envision study.
The situation underscores the complex balance between advancing potentially transformative treatments and ensuring patient safety in the development of gene therapies. While Elevidys has shown promise in treating Duchenne muscular dystrophy, this recent setback highlights the ongoing challenges faced by companies developing innovative genetic treatments for serious medical conditions.
The temporary suspension of these trials represents a cautious approach by regulatory authorities and pharmaceutical companies as they work to thoroughly investigate safety concerns while continuing to advance potential treatments for patients with this devastating muscle-wasting condition. The outcome of the investigation into this patient death could have significant implications for the future development and implementation of gene therapies in the treatment of Duchenne muscular dystrophy.