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 thorough investigation into the cause of death.
The affected patient experienced acute liver failure and had a recent cytomegalovirus infection that may have played a role in the outcome. While liver injury is a recognized potential side effect of Elevidys and similar gene therapies, this marks the first fatal case of acute liver failure among more than 800 patients who have received the treatment.
Three clinical trials have been impacted by the suspension, including the crucial global Phase 3 Envision study examining the therapy’s effects in both ambulatory and non-ambulatory patients aged 8 to 17. The pause affects only new patient enrollment and dosing; previously treated participants will continue to be monitored for data collection purposes.
The development represents another challenge for Elevidys, which received initial FDA approval in 2023 for a limited group of Duchenne patients before gaining expanded authorization the following year, despite mixed clinical trial results. The therapy is currently approved in the United States for patients four years and older with specific gene mutations, with full approval for ambulatory patients and accelerated approval for non-ambulatory individuals.
The news has significantly impacted Sarepta Therapeutics, which developed Elevidys and licensed its European rights to Roche. Following the March 18 announcement of the patient death, Sarepta’s stock price declined sharply, falling an additional 7% to
approximately $58 per share in early trading on Thursday, amid broader market concerns over President Trump’s tariff announcement. The company’s shares had previously traded above $100 before the news broke.
Industry analysts, including Leerink Partners’ Joseph Schwartz, suggest that while the therapy’s reputation has suffered, investors may be overreacting to the setback. The clinical trial suspension could delay study results and impact the ongoing European regulatory review, but analysts note that sales outside the United States would represent a relatively small portion of Sarepta’s revenue stream.
The Envision trial, which was expected to produce results in 2027, is particularly significant as its findings are required to confirm the accelerated approval granted for non-ambulatory patients in the United States. The study’s temporary suspension adds uncertainty to this timeline.
Roche’s decision to halt the trials reflects the careful approach regulatory authorities and pharmaceutical companies must take when investigating serious adverse events in gene therapy trials. The suspension will remain in place until researchers and regulators complete their analysis of the patient death and determine appropriate next steps for the clinical program.
This development occurs against the backdrop of growing interest and investment in gene therapies for rare diseases, highlighting the complex balance between advancing potentially transformative treatments and ensuring patient safety. The outcome of the
investigation could have broader implications for the gene therapy field, particularly regarding liver toxicity monitoring and management in similar treatments.
While the immediate impact has been significant for Sarepta’s market value, the long-term implications for Elevidys will likely depend on the investigation’s findings and any resulting modifications to the therapy’s safety protocols or administration guidelines.