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

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: Market Potential and Financial Outlook - If approved, trofinetide's launch in the EU is expected to enhance Acadia's growth prospects [6]. - Acadia anticipates generating over $1 billion in combined net sales for Nuplazid and Daybue in 2025 [8]. - The company is also evaluating other pipeline candidates, including ACP-101 for Prader-Willi Syndrome and ACP-204 for Alzheimer's disease psychosis, with key data expected in 2026 [9][10]. Group 3: Stock Performance - Acadia's shares have decreased by 41.6% over the past year, compared to a 14.8% decline in the industry [3].