Roche is taking another chance on an embattled gene therapy for Duchenne muscular dystrophy, announcing on Thursday a new trial that could support a reworked approval submission in Europe.
Roche owns rights outside the U.S. to Elevidys, which was developed by Sarepta Therapeutics and is approved for use in nine countries as a treatment for certain Duchenne patients who can still walk. Europe’s main drug regulator, however, issued a negative opinion on Elevidys last year. It noted how testing didn’t show a meaningful benefit on motor function and that the therapy’s principal biological effect — producing a miniature version of a muscle-protecting protein — couldn’t be linked to a positive impact.
That setback was one of several in 2025 for Elevidys. The treatment was the first of its kind to win a regulatory approval and the culmination of years of scientific research. But clearance in the U.S. was based on mixed study results that cast doubt on its benefits. Last summer, it was embroiled in controversy after being linked to the deaths of two drug recipients. Elevidys was temporarily removed from the market in the U.S. and, since then, the Food and Drug Administration has restricted its use in certain people. Testing in Europe was temporarily halted as well.
Along the way, Sarepta laid off staff and halted much of its gene therapy work. It also significantly dialed back Elevidys’ once multibillion-dollar sales expectations. The therapy generated around $900 million in net product revenue in 2025, but sales have been consistently declining. Wall Street analysts expect the therapy’s 2026 numbers to fall well short of the $500 million mark Sarepta has predicted.
Roche’s new trial might eventually help change this trajectory. Following the negative decision from the European Medicines Agency last year, the Swiss drugmaker said it intended to work with the regulator to “explore a potential path forward” for Elevidys in Europe. On Thursday, Roche revealed that those talks have led to a new global Phase 3 trial that could generate the kind of data required for a resubmission.
According to Roche, the study will enroll about 100 “early ambulatory” patients with Duchenne and randomize them to receive either Elevidys or a placebo. The study’s main objective is to prove treatment can lead to a meaningful change over 72 weeks in how quickly someone can get up from the floor, a measure known as “time to rise” that’s an important predictor of disease progression.
Notably, the trial is longer and designed differently than a previous placebo-controlled test that produced underwhelming results. Roche, in a statement, said it hopes the study will “strengthen the robust body of clinical evidence” for Elevidys and enable not only new approvals, but reimbursement for the therapy in additional countries.
“Our confidence is rooted in robust long-term data showing the durable efficacy and safety of Elevidys, alongside the experience of treating more than a thousand ambulatory boys worldwide,” Chief Medical Officer Levi Garraway said in the statement.
The announcement represents a “positive” for Sarepta, in that Roche “remains committed” to an approval in Europe, wrote Leerink Partners analyst Joseph Schwartz in a Thursday client note. But it also makes clear there is “no path forward based on the current data,” and a lengthy timeline to market there, he added.
Schwartz anticipates the study will take around a year and a half to enroll and produce results in 2028.
