A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Latest Information Update: 23 Apr 2024
At a glance
- Drugs Trofinetide (Primary)
- Indications Rett syndrome
- Focus Registrational; Therapeutic Use
- Acronyms LAVENDER
- Sponsors Acadia Pharmaceuticals
- 22 Apr 2024 According to an Acadia Pharmaceuticals media release, company announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadias submission. The Health Canada filing is supported by results from the positive pivotal Phase 3 LAVENDER study.
- 12 Sep 2023 Results assessing Population Pharmacokinetic Modelling to Confrm Weight-Based Banded Dosing & Exposure-Response Ef- ficacy Analyses to Support Trofnetide Treatment in Rett Syndrome using this study and other 13 studies presented at the 2023 American College of Clinical Pharmacology Annual Meeting
- 12 Sep 2023 Results assessing Population Pharmacokinetic Modelling to Predict Trofnetide Exposure & Exposure-Response Safety Analyses in Girls With Rett Syndrome Aged 2 to 4 Years presented at the 2023 American College of Clinical Pharmacology Annual Meeting