The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US teenager, who suffered acute liver failure after receiving Elevidys, can be precisely determined.
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.
