ACAD Seeks EU Nod for Rett Syndrome Drug, Outlines 2025 Pipeline Goals

Core Viewpoint - Acadia Pharmaceuticals has submitted a marketing authorization application to the European Medicines Agency for trofinetide to treat Rett syndrome, with an expected approval in Q1 2026, potentially making it the first approved therapy for this condition in the EU [1][2]. Group 1: Product Development and Approval - Trofinetide is already approved in the U.S. under the name Daybue for treating Rett syndrome in patients aged two years and older, launched in April 2023 [2]. - The MAA for trofinetide in the EU is based on positive results from the phase III LAVENDER study, which involved 187 female patients aged 5 to 20 years [4]. - The LAVENDER study met its co-primary endpoints, showing significant improvements in the Rett Syndrome Behaviour Questionnaire total score and the Clinical Global Impression-Improvement scale score at week 12 [5]. Group 2: Financial Outlook and Pipeline - Acadia expects to generate over $1 billion in combined net sales for Nuplazid and Daybue in 2025 [8]. - The company is evaluating ACP-101 in the phase III COMPASS PWS study for Prader-Willi Syndrome, with top-line data expected in the first half of 2026 [8]. - Acadia is also developing ACP-204 in the phase II RADIANT study for Alzheimer's disease psychosis, with top-line data expected in mid-2026 [9].