One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the death of a U.S. teenager, a data monitoring committee concluded that they should continue unchanged.
The holds placed in the EU on three clinical trials for Sarepta and Roche’s gene therapy Elevidys may soon lift after a data monitoring committee determined that the benefit-risk ratio favors their continuation without changes.
On March 18th, Sarepta announced that a patient who had been treated with Elevidys had died of acute liver failure. According to a statement released by Sarepta on Friday, an independent data monitoring committee (DMC) convened Thursday, the same day the holds were announced. EU member states had requested the committee meet to review the death.
The committee “concurred that based on the totality of evidence, the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols.”
Sarepta and Roche plan to submit the committee’s conclusion to EU regulators within a week and Sarepta said Friday it does not anticipate a “material impact” on the timeline for the affected trials.
Original story published April 3, 2025.
Sarepta’s Duchenne Gene Therapy Placed on Hold in Europe Pending Death Investigation
The European Medicines Agency has requested that Sarepta Therapeutics and partner Roche pause a trio of clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys in response to a patient death that occurred outside the scope of the studies.
In a letter addressed to the World Duchenne Organization and dated March 31, Roche shared that it was pausing Phase I, Phase II and Phase III trials all studying the gene therapy for treating Duchenne muscular dystrophy (DMD).
The European Medicines Agency (EMA) asked for the trials to be paused until the exact cause of death of a U.S. teenager, who had suffered acute liver failure (ALF) after taking Elevidys, can be precisely determined. Sarepta announced last month at the time of the patient’s death that it would update Elevidys’ label to reflect the safety signal.
Analysts at BMO were nonplussed by the trial pause. “We view the requested clinical hold as an administrative step, pending [data monitoring committee] meeting, that can be resolved within ~6mo given Elevidys benefit/risk in the context of DMD being a universally fatal disease,” the analysts wrote in a note late Wednesday (emphasis theirs).
Other analysts were more mixed. “On one hand, the risk of fatal ALF is rare (0.125%) and our experts affirm the news is unsurprising to them. However, we’ve felt the death should not be fully dismissed unless [Sarepta management] can provide color on some outstanding questions,” Jefferies analysts wrote Wednesday.
The connection between biopharmas working on DMD and the patient community is especially tight knit. Following the patient’s death, Sarepta sent a letter directly to the DMD patient community, pledging transparency and sharing data as the company investigates the death.
The sentiment mirrors those reflected in Roche’s letter earlier this week to the DMD community. The company signed off saying, “We truly appreciate your leadership and the opportunity to work together with you in these efforts.”
The patient death after taking Elevidys is one of a series of deaths from DMD gene therapies. Two patients died, one in 2021 and another in 2024, while taking Pfizer’s investigational gene therapy fordadistrogene movaparvovec. That program was later scrapped. In 2022, a CRISPR-based Duchenne therapy led to another patient death, potentially caused by the viral vector used.
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