Roche Faces Setback for DMD Gene Therapy Elevidys in Europe, Commits to Path Forward

Roche, the Swiss pharmaceutical giant, has encountered a significant obstacle in its efforts to bring the gene therapy Elevidys to European patients with Duchenne muscular dystrophy (DMD). Despite this setback, the company remains committed to finding a way forward for the treatment in the European Union.

EMA's CHMP Recommends Against Elevidys Approval

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended against granting conditional marketing authorization for Elevidys in Europe. The therapy, developed by Sarepta Therapeutics and marketed by Roche outside the United States, was intended for ambulatory DMD patients between the ages of 3 and 7 years.

The EMA's decision was based on data from the main study supporting Roche's approval bid, which "failed to show that Elevidys had an effect on movement abilities after 12 months." The regulator noted that the difference in functional motor improvement scores between Elevidys patients and those on placebo was "not statistically significant."

Dr. Levi Garraway, Roche's chief medical officer and head of global product development, expressed disappointment with the CHMP's negative opinion, citing the "urgent need for disease-modifying therapies for children in the EU living with Duchenne."

Roche's Response and Future Plans

Despite the setback, Roche remains confident in Elevidys' potential value for ambulatory patients. The company plans to continue working with the EMA to "explore a potential path forward" for the gene therapy in Europe.

Roche maintains that while the Embark study failed to meet its primary endpoint measuring effect on movement ability, Elevidys demonstrated "clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures" compared to placebo. The company also cited longer-term efficacy data from Embark and a three-year pooled efficacy analysis from three separate Elevidys studies, which "showed clinically meaningful improvements across key measures of motor function."

Safety Concerns and Global Impact

The European setback comes amid mounting safety concerns over Sarepta's gene therapy platform. Two patient deaths from acute liver failure have been reported this year, leading to a pause in commercial shipments of Elevidys to non-ambulatory patients in the U.S. in June. A third death was recently confirmed in an older patient with a different form of muscular dystrophy who received an investigational Sarepta gene therapy using the same viral vector as Elevidys.

In response to these safety issues, the FDA requested that Sarepta voluntarily pause shipments of Elevidys to all commercial patients in the United States. Roche has also paused Elevidys shipments in countries outside the U.S. where approvals were based on the FDA's authorizations, primarily affecting Gulf Region states such as the United Arab Emirates and Qatar.

Despite these challenges, Roche remains committed to the potential of gene therapy in treating human health. Teresa Graham, CEO of Roche Pharma, acknowledged the complexities of gene therapy development but expressed hope for finding a path forward for Elevidys, emphasizing the need for discussions with regulators about safety while maintaining belief in the product's risk-benefit profile.