The Brazilian Health Regulatory Agency (Anvisa) announced that it has temporarily suspended the marketing, distribution, manufacturing, import, advertising, and use of the gene therapy drug Elevidys (delandistrogen moxeparvovek) in Brazil as a precautionary measure. The suspension will remain in effect until recent safety uncertainties related to fatal cases of acute liver failure in patients treated with the product in the United States are fully clarified.
This decision was made in light of new regulatory information published by the U.S. Food and Drug Administration (FDA) reporting three deaths associated with the use of gene therapy products featuring AAVrh74 vector technology developed by Sarepta Therapeutics.
Two cases involved non-ambulatory pediatric patients with Duchenne Muscular Dystrophy (DMD) treated with Elevidys, and the third case involved an adult patient with Limb-Girdle Muscular Dystrophy who was receiving an investigational gene therapy product based on the same viral vector. Both events involved progression to acute liver failure with a fatal outcome.
Learn More: Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States
Learn More: The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys
