Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD)

Today, Roche revealed encouraging topline data from the second year of the EMBARK trial, a worldwide, double-blind, randomised phase III investigation of Elevidys (delandistrogene moxeparvovec), the first gene therapy to be authorized for the treatment of Duchenne muscular dystrophy.

When compared to a predetermined propensity-weighted untreated external control group, statistically significant and clinically meaningful improvements were seen in three important motor function tests two years following Elevidys treatment: NSAA, TTR, and 10MWR. Between one and two years following dose, the functional differences between those receiving Elevidys and those in the external control group grew. When taken as a whole, these findings show that Elevidys has consistently benefited.

“After two years of treatment with Elevidys, we are seeing multiple sustained benefits in the day-to-day lives of these young boys, all of which are indicators of its disease modifying potential in Duchenne,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “These results, which include improvements in standing, walking and running, represent meaningful progress and we plan to share them with health authorities as quickly as possible.”

Read More: Frequently Asked Questions About Elevidys

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