Sarepta’s Duchenne Gene Remedy Positioned on Maintain in Europe Pending Dying Investigation

The European Medicines Company has requested that Sarepta Therapeutics and associate Roche pause a trio of scientific trials of their Duchenne muscular dystrophy gene remedy Elevidys in response to a affected person demise that occurred outdoors the scope of the research.

In a letter addressed to the World Duchenne Group and dated March 31, Roche shared that it was pausing Part I, Part II and Part III trials all finding out the gene remedy for treating Duchenne muscular dystrophy (DMD).

The European Medicines Company (EMA) requested for the trials to be paused till the precise explanation for demise of a U.S. teenager, who had suffered acute liver failure (ALF) after taking Elevidys, will be exactly decided. Sarepta introduced final month on the time of the affected person’s demise that it might replace Elevidys’ label to mirror the security sign.

Analysts at BMO had been nonplussed by the trial pause. “We view the requested scientific maintain as an administrative step, pending [data monitoring committee] assembly, that may be resolved inside ~6mo given Elevidys profit/danger within the context of DMD being a universally deadly illness,” the analysts wrote in a observe late Wednesday (emphasis theirs).

Different analysts had been extra blended. “On one hand, the danger of deadly ALF is uncommon (0.125%) and our specialists affirm the information is unsurprising to them. Nevertheless, we’ve felt the demise shouldn’t be totally dismissed except [Sarepta management] can present colour on some excellent questions,” Jefferies analysts wrote Wednesday.

The connection between biopharmas engaged on DMD and the affected person neighborhood is particularly tight knit. Following the affected person’s demise, Sarepta despatched a letter on to the DMD affected person neighborhood, pledging transparency and sharing knowledge as the corporate investigates the demise.

The sentiment mirrors these mirrored in Roche’s letter earlier this week to the DMD neighborhood. The corporate signed off saying, “We actually respect your management and the chance to work along with you in these efforts.”

The affected person demise after taking Elevidys is one among a sequence of deaths from DMD gene therapies. Two sufferers died, one in 2021 and one other in 2024, whereas taking Pfizer’s investigational gene remedy fordadistrogene movaparvovec. That program was later scrapped. In 2022, a CRISPR-based Duchenne remedy led to a different affected person demise, doubtlessly attributable to the viral vector used.