Roche initiates a Phase III trial of Elevidys to support European Medicines Agency resubmission and expand access for Duchenne muscular dystrophy patients.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Hoffmann-La Roche Ltd has announced plans to initiate a new global Phase III study of Elevidys, a gene therapy approved for treating the underlying cause of Duchenne muscular dystrophy (DMD). The study is intended to generate additional data to support regulatory resubmission to the European Medicines Agency and expand patient access in Europe and other regions.
The planned trial follows feedback from the European Medicines Agency and input from the Duchenne community, with a focus on addressing unmet needs among ambulatory boys living with DMD.
The pivotal study will evaluate both efficacy and safety of Elevidys compared with placebo over a 72-week period. Approximately 100 early ambulatory boys with DMD will be enrolled.
The primary endpoint is change in “Time to Rise” (TTR) velocity from the floor, a clinically relevant measure associated with disease progression. Patients initially assigned to placebo will have the opportunity to receive Elevidys after completion of the primary study period.
Elevidys is the first approved disease-modifying gene therapy for DMD. It works by enabling expression of a micro-dystrophin protein in skeletal, respiratory, and cardiac muscle, targeting the root cause of the disease.
The therapy is administered as a one-time intravenous infusion and has been studied in a broad clinical development program with follow-up extending up to six years. To date, more than 1,200 patients with DMD have received Elevidys across clinical trials and real-world settings.
Elevidys is currently approved in nine countries, including the United States, Japan, and several countries in the Middle East and Latin America, for ambulatory patients with confirmed DMD mutations.
According to Levi Garraway, the initiation of the study reflects Roche’s continued commitment to the Duchenne community and its goal of making the therapy broadly accessible. He emphasized that the company’s confidence is supported by long-term data demonstrating sustained efficacy and safety, along with real-world treatment experience in a large patient population.
Safety considerations have played a key role in shaping the development and regulatory pathway of Elevidys, with liver-related adverse events prompting label updates and stricter risk management requirements across regulatory jurisdictions. Reports of serious adverse events, including patient deaths in the broader Duchenne gene therapy setting, have further intensified oversight and led to more cautious regulatory approaches. These factors have also driven additional clinical investigation, including studies such as the ENDEAVOUR cohort 8 evaluating the use of sirolimus before and after gene therapy administration to help manage immune-mediated toxicities.
Sarepta Therapeutics is responsible for regulatory approval and commercialization of Elevidys in the United States, as well as manufacturing. Roche oversees regulatory and commercial activities outside the U.S., while Chugai Pharmaceutical manages commercialization in Japan.
Duchenne muscular dystrophy is a rare, progressive genetic disorder characterized by muscle degeneration and reduced life expectancy. It primarily affects boys and places a significant burden on patients, families, and healthcare systems.
Through this additional Phase III study, Roche aims to strengthen the clinical evidence base for Elevidys and support broader regulatory approvals and reimbursement decisions globally, with the ultimate goal of expanding access to eligible patients.
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About the Writer
Chikkula Pavan Kumar, PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.