CHMP Issues Negative Recommendation on Sarepta’s Elevidys

Rare Daily Staff

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended that conditional approval not be granted for Elevidys, Sarepta’s Duchenne muscular dystrophy gene therapy developed in partnership with Roche in Europe.

Roche is responsible for regulatory approvals and marketing of Elevidys outside the United States. Roche said it will continue discussions with the EMA to explore possible pathways to make the gene therapy available to individuals living with Duchenne muscular dystrophy (DMD) in the European Union. Roche is seeking conditional approval to market Elevidys for ambulatory patients aged 3 to 7 years with the rare neuromuscular condition.

This decision marks the latest setback for Sarepta, following the deaths of two non-ambulatory patients from acute liver failure after treatment with Elevidys, as well as the death of a third patient with limb-girdle muscular dystrophy treated with a different Sarepta gene therapy that uses the same serotype.

Elevidys has been approved in the United States for ambulatory patients with Duchenne muscular dystrophy. Sarepta has voluntarily withdrawn the therapy from the U.S. market at the request of the U.S. Food and Drug Administration while safety risks are further evaluated.

The CHMP decision was based on data from the pivotal phase 3 EMBARK study, which failed to meet its primary endpoint after one year. Nevertheless, Roche reported that Elevidys demonstrated clinically meaningful and statistically significant improvements in secondary endpoints assessing functional outcomes compared to placebo. Roche provided additional efficacy data to the EMA, including two-year results from the EMBARK study and a three-year pooled efficacy analysis from three other Elevidys studies, all showing meaningful improvements in key motor function measures.

“We are disappointed by the CHMP’s negative opinion, given the urgent need for disease-modifying therapies for children in the EU living with Duchenne,” said Levi Garraway, chief medical officer and head of global product development at Roche. “With an average life expectancy of only 28 years, achieving disease stabilization is a major advance for individuals living with Duchenne, their families, and caregivers. We are confident in the value Elevidys can bring to ambulatory patients.”

Photo: Levi Garraway, chief medical officer and head of global product development at Roche