Daybue

The approval was supported by data from the randomized, double-blind, placebo-controlled phase 3 LAVENDER study (ClinicalTrials.gov Identifier: NCT04181723), which evaluated the efficacy and safety of trofinetide in female patients 5 to 20 years of age with Rett syndrome. Patients were randomly assigned to receive trofinetide (n=93) or placebo (n=94) orally or via gastrostomy tube twice daily for 12 weeks. 

The coprimary endpoints were the change from baseline to week 12 in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score (a caregiver assessment) and the Clinical Global Impression-Improvement (CGI-I) score (a physician assessment). Lower scores reflect lesser severity in signs and symptoms of Rett syndrome.

Treatment with trofinetide was associated with a statistically significant improvement on the RSBQ compared with placebo (-4.9 vs -1.7, respectively; treatment difference, -3.2 [95% CI, -5.7, -0.6]; P =.018). A statistically significant improvement over placebo was also observed on the CGI-I (3.5 vs 3.8, respectively; treatment difference, -0.3 [95% CI, -0.5, -0.1]; P =.003).