• Health Canada has approved Daybue (trofinetide) for treating Rett syndrome in patients aged two years and older, marking the first approved treatment for the condition in Canada.
• The approval was based on Phase 3 LAVENDER study results, which demonstrated significant improvements in behavior and communication compared to placebo over a 12-week period.
• Daybue, developed by Acadia Pharmaceuticals, is a synthetic analog of insulin-like growth factor 1, administered orally to lessen inflammation and promote nerve cell connections.
• Experts welcome Daybue as a crucial advancement, offering a specific therapeutic option beyond symptomatic management for Rett syndrome, a neurodevelopmental disorder affecting primarily females.