Roche said that it is initiating a late-stage clinical trial of its gene therapy Elevidys for Duchenne muscular dystrophy (DMD) after receiving feedback from the European Medicines Agency (EMA). The therapy, developed in partnership with Sarepta Therapeutics, previously failed to gain support from the EMA for use in ambulatory patients.
The new phase III study is intended to generate additional placebo-controlled data required for a regulatory resubmission to the EMA. The trial will run for more than 72 weeks and include around 100 early ambulatory boys diagnosed with DMD. It will evaluate the efficacy and safety of Elevidys compared with a placebo. The primary endpoint is the change in “time to rise” from the floor velocity, a measure used to assess disease progression.
Duchenne muscular dystrophy is a rare, inherited condition that mainly affects boys and causes progressive muscle weakening over time. Elevidys is currently approved in nine countries for ambulatory boys with a confirmed DMD mutation, including the United States. In the U.S., shipments of the therapy were temporarily paused last July.
Roche is initiating the study following feedback from the EMA after the agency previously refused to support approval of Elevidys for ambulatory patients. In its earlier assessment, the EMA noted that the therapy’s 0.65-point difference compared with placebo in the 34-point North Star Ambulatory Assessment was not statistically significant.
In the United States, Elevidys was approved based on results from the phase III Embark trial. That study did not meet its primary endpoint, failing to show a statistically significant improvement in motor function compared with placebo after one year. Sarepta has cited longer-term findings, including three-year results showing a 4.39-point improvement compared with a propensity-weighted untreated external control group.
The new trial will again compare Elevidys with a placebo but over a longer duration. Participants initially assigned to the placebo group will be eligible to receive the therapy after the primary 72-week study period.
Reports of two deaths involving severe liver injury in non-ambulatory patients led to restrictions on the therapy’s use. Roche halted distribution of Elevidys for non-ambulatory patients in territories outside the United States and later paused shipments completely in some countries. In the U.S., the therapy remains available but is limited to ambulatory patients.
Roche stated that its current focus is on the ambulatory population, where available data indicate a positive benefit-risk profile and a durable treatment effect. Additional research is ongoing to address safety considerations in non-ambulatory patients, including Sarepta’s testing of an enhanced immunosuppression regimen. Results from cohort 8 of the Endeavor trial are expected to inform any future plans for that group.
“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,” said Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development.
The company said the findings from the new study are expected to support a future resubmission to European regulators for the ambulatory DMD population.
Why Roche Relaunched the Elevidys Trial
The decision to launch a new study for Elevidys comes after the EMA previously declined approval, citing insufficient evidence of clinical benefit. Regulators noted that earlier trials did not show statistically significant improvements in motor function compared to placebo.
In response, Roche is redesigning its approach to generate stronger, placebo-controlled data that can better demonstrate the effectiveness of Elevidys.
Details of the New Phase III Study
The new global Phase III trial for Elevidys will enroll approximately 100 early ambulatory boys with DMD. The study will compare Elevidys against a placebo over a 72-week period, focusing on both safety and efficacy.
The primary endpoint will measure changes in “time to rise” from the floor, a clinically meaningful indicator of disease progression. This refined study design aims to directly address the concerns raised by the EMA.
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