SHERIDAN, WYOMING – July 29, 2025 – Roche’s gene therapy for Duchenne muscular dystrophy (DMD), Elevidys™ (delandistrogene moxeparvovec), has encountered a regulatory hurdle in the European Union, following a negative opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP). While disappointing, the decision does not mark the end of the road for the therapy, which remains the first and only disease-modifying gene therapy available for DMD and continues to show promise in stabilizing disease progression in ambulatory patients.
First-in-class gene therapy meets complex regulatory path
The CHMP’s negative stance was issued in response to Roche’s application for conditional marketing authorization (CMA) of Elevidys for ambulatory children aged 3–7 with DMD. Roche has confirmed that it will continue dialogue with the EMA to explore a viable regulatory pathway.
Despite this setback, Elevidys remains a pioneering treatment. It is designed as a one-time intravenous infusion targeting skeletal, cardiac, and respiratory muscles, delivering a shortened dystrophin gene — a critical protein absent in DMD patients. The gene therapy has already been approved in nine countries and administered to over 900 patients globally, including more than 760 ambulatory individuals.
Clinical data support stabilization despite missed primary endpoint
The CHMP’s decision followed a review of data from the pivotal Phase III EMBARK study — the largest DMD gene therapy program to date. While the primary endpoint was not met at the one-year mark, Elevidys demonstrated clinically meaningful and statistically significant improvements across multiple secondary endpoints.
Additional efficacy data submitted to the EMA included:
- Two-year results from EMBARK
- A three-year pooled efficacy analysis from three other Elevidys studies
- Published one-year findings in Nature Medicine (October 2024)
- Presentation of year-two results at the 2025 MDA Clinical Conference in Dallas
These long-term data points consistently indicate a stabilizing effect on motor function and a manageable safety profile in ambulatory DMD patients.
Real-world urgency underscores need for earlier intervention
Duchenne muscular dystrophy is a rare and devastating X-linked neuromuscular disorder caused by mutations in the DMD gene, affecting approximately 1 in 5,000 boys worldwide. Without functional dystrophin, muscles deteriorate rapidly, leading to irreversible damage, loss of ambulation, respiratory failure, and cardiac complications. Average life expectancy is just 28 years.
Elevidys offers a potential shift in care by slowing this trajectory — especially when administered before irreversible muscle loss. Roche’s Chief Medical Officer, Dr. Levi Garraway, underscored the human cost of delay: “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. With an average life expectancy of only 28 years, achieving disease stabilisation 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.”
Safety remains a focal point amid broader rollout
While Elevidys has shown no treatment-related fatalities among ambulatory patients, safety concerns have arisen in non-ambulatory populations. In June, Roche implemented dosing restrictions for non-ambulatory individuals following two reported fatalities. On July 22, Roche temporarily paused new orders outside the U.S. where local approvals are FDA-referenced, pending further safety evaluations.
These steps reflect an abundance of caution and underscore Roche’s emphasis on patient safety. The company has reaffirmed that, “based on the totality of available data,” the benefit-risk profile remains positive for ambulatory individuals.
Strategic outlook for hospitals, providers, and global regulators
For hospitals and specialists, the availability of a gene therapy like Elevidys could transform DMD care models. As a one-time treatment, it may reduce long-term care burdens, improve mobility outcomes, and lessen respiratory and cardiac complications — translating into both quality-of-life improvements and systemic cost offsets.
The EMA’s decision is not final, and continued discussions between Roche and European regulators may yet lead to a revised submission pathway. Meanwhile, real-world data from approved markets and emerging long-term evidence will be critical in shaping future access decisions.
Learn more about Elevidys and Roche’s neuromuscular innovation at www.roche.com.