Roche announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion on the conditional marketing authorisation (CMA) application for Elevidys (delandistrogene moxeparvovec). The application was for use in ambulatory children aged 3 to 7 years with Duchenne muscular dystrophy (DMD). Roche plans to continue working with the EMA to explore possible next steps, given the urgent need for new treatment options.

“We are disappointed by the CHMP’s decision, especially given the critical need for disease-modifying treatments for children in the EU living with Duchenne,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development at Roche. “With a life expectancy of just 28 years, stabilising disease progression is a meaningful advance for patients and their families. We believe Elevidys can provide real value to ambulatory patients.”

The CHMP opinion was based on data from the most extensive gene therapy program in DMD to date, including the Phase III EMBARK study. While EMBARK did not meet its primary endpoint after one year, Elevidys showed statistically significant and clinically meaningful improvements in key secondary functional outcomes compared to placebo. The safety profile was consistent and manageable.

Duchenne is a rare, genetic muscle-wasting disease that begins in early childhood and progresses quickly. Everyone affected will eventually lose the ability to walk and will experience serious decline in upper limb, heart, and lung function. Average life expectancy is 28 years. The impact on patients, families, and caregivers is severe—physically, emotionally, and financially.

Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne and has consistently shown it can stabilise or slow disease progression, with long-lasting effects on muscle health and function.