Roche announces EMA has initiated review of the Elevidys Marketing Authorisation application for the treatment of Duchenne muscular dystrophy (DMD)

Roche has announced that the European Medicines Agency (EMA) is reviewing the marketing authorization application for Elevidys, a gene therapy for Duchenne muscular dystrophy in ambulatory patients aged 3-7. Duchenne is a severe muscle-wasting disease with no cure, making new treatments crucial. The application is based on the Phase 3 EMBARK study, which showed that Elevidys provides clinically meaningful benefits despite not meeting its primary endpoint. Elevidys-treated patients demonstrated significant improvements in mobility tests and stride velocity, a novel digital endpoint. Roche is also conducting additional studies to expand Elevidys' label to include younger and older patients. Through a collaboration with Sarepta, Roche is responsible for Elevidys' availability outside the US, where it's already approved in several countries. Elevidys is a one-time gene therapy designed to address the underlying cause of Duchenne by targeting muscle expression of dystrophin. Duchenne affects approximately 1 in 5,000 boys, causing progressive muscle degeneration and significantly impacting life expectancy and quality. Roche is dedicated to developing innovative treatments for neurological disorders, including Duchenne muscular dystrophy, to improve patients' lives.