Elevidys Gene Therapy Faces Setback in Europe

While Elevidys, a gene therapy for Duchenne muscular dystrophy, has brought hope to many, recent setbacks have been felt deeply by families in Ireland and around the world.

On Friday, 24 July, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a recommendation not to approve Elevidys for use in Europe.

Roche, the company that applied for approval, can ask for a re-examination of the opinion within the next 15 days. Roche said that it would work with the EMA on a potential path forward for Elevidys in Europe. Click here to read their full release.

In making its decision, the EMA said that the study did not show a clear improvement in movement abilities 12 months after treatment with Elevidys. The difference in motor function between patients who received the therapy and those given a placebo was not statistically significant, meaning the observed results could have been due to chance. While many patients treated with Elevidys did produce a shortened form of the dystrophin protein, the EMA concluded that the level of this protein could not be linked to an improvement in movement abilities.

Roche also presented data for a subgroup of patients who seemed to respond better to Elevidys; however, the EMA found that the evidence was still not strong enough to demonstrate the therapy’s effectiveness. Click here to read the EMA opinion.

This news comes after the U.S. Food and Drug Administration (FDA) asked Sarepta to pause all shipments of Elevidys in the United States because of serious safety concerns, including reports of life-threatening liver complications. However, on Monday, July 28, the FDA announced that Elevidys can now be shipped again, but only for boys with Duchenne who are still able to walk.

We know this news from the EMA will be disappointing for many families who are affected by Duchenne. We will continue to follow developments closely and share any further updates as they become available. Our thoughts are with everyone impacted during this uncertain time.