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Roche: Updates on Gene Therapy Delandistrogene Moxeparvovec (Elevidys™) Program in Recent April 2024 Community Letter

May 3, 2024

Defeat Duchenne Canada is pleased to present the most recent developments from Roche regarding advancements in the gene therapy delandistrogene moxeparvovec (Elevidys™) program. This update encompasses crucial information concerning their application to the European Medicines Agency (EMA) and the initiation of two clinical trials outside of the United States. They have included these significant strides towards improving treatment options for individuals affected by Duchenne muscular dystrophy in their April 2024 Community Letter.

Read the full update below:

EMBARK Phase 3 Updates

The EMBARK study continues to conduct additional analyses on the one-year data and anticipates completion of Part 2 with two-year data. An application to EMA is planned for this year.

At this time, delandistrogene moxeparvovec is approved in the US. The FDA is reviewing an application to potentially expand access to a broader population in the US.

ENVISION Updates

ENVISION is a Phase 3 study that investigates the safety and efficacy of delandistrogene moxeparvovec in participants who are ambulatory (aged 8 to under 18 years old) and non-ambulatory (no age limitation).

The study aims to enroll 148 participants and all will receive the gene therapy over the course of the multi-year study. Approximately 50 sites are planned outside the US; the study has now opened in five countries (Belgium, Italy, Japan, Spain and the UK) with additional countries soon to open. Currently, there are no sites announced in Canada.

ENVOL Updates

ENVOL (NCT06128564) is a Phase 2 study evaluating the safety of delandistrogene moxeparvovec and expression of delandistrogene moxeparvovec micro-dystrophin in young children, including babies and newborns.

This study aims to enroll 21 participants under 4 years of age. All participants will be part of the study for five years after receiving the gene therapy. The study has opened in three countries (Germany, Italy and Spain) with additional countries soon to open. Locations will be posted on clinicaltrials.gov when they are open or nearly open for enrollment. Currently, there are no sites announced in Canada.

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