The U.S. Food and Drug Administration has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older.
Approval was based on results from the phase 3 LAVENDER study in which the efficacy and safety of Daybue was assessed versus placebo in 187 female patients with Rett syndrome (ages 5 to 20 years). Compared with placebo, Daybue was associated with statistically significant improvement for change from baseline in the Rett Syndrome Behaviour Questionnaire total score and the Clinical Global Impression-Improvement scale score at week 12.
