Roche plans to initiate a new global, pivotal Phase 3 study of Elevidys (delandistrogene moxeparvovec), a gene therapy approved in several countries for ambulatory boys with Duchenne muscular dystrophy (DMD). The trial is intended to generate additional placebo-controlled data to support a regulatory resubmission to the European Medicines Agency and to expand access for ambulatory patients in Europe and other regions.
According to Roche, Elevidys has shown the ability to slow disease progression in ambulatory boys with DMD based on six years of clinical data, including the EMBARK Phase 3 study. Elevidys is currently approved in nine countries for ambulatory boys with a confirmed DMD mutation.
The new Phase 3 study will enroll approximately 100 early ambulatory boys with DMD and evaluate the efficacy and safety of Elevidys compared with placebo over 72 weeks. The primary endpoint is change in “Time to Rise” from the floor velocity, a functional measure associated with future disease progression. After the 72‑week primary period, participants initially assigned to placebo will be eligible to receive Elevidys.
Roche stated that the study is designed to build on existing long-term data and to support future regulatory and reimbursement applications aimed at broadening access to Elevidys for ambulatory DMD patients in additional countries.
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