Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU

Roche announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization for Elevidys, a gene therapy for ambulatory individuals with Duchenne muscular dystrophy (DMD). Roche plans to continue working with the EMA to explore a potential path forward for Elevidys. The CHMP opinion is based on data from the largest and broadest gene therapy clinical program in DMD to date, including results from the pivotal Phase III EMBARK study. Elevidys showed sustained stabilization or slowing of disease progression and a consistent and manageable safety profile in ambulatory patients. Despite the negative opinion, Roche believes the benefit-risk profile remains positive in the ambulatory patient population. Elevidys is the first and only approved gene therapy targeting the underlying cause of DMD, consistently demonstrating stabilization or slowing of disease progression. DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood, with an average life expectancy of only 28 years. Roche recognizes the significant unmet medical need for those living with DMD and is actively working with health authorities across the globe to bring Elevidys to patients and their families as soon as possible. To date, more than 900 individuals with DMD, including 760 ambulatory patients, have been treated with Elevidys in clinical and real-world settings.