• Acadia plans to request re-examination of CHMP opinion on trofinetide for Rett syndrome
• Trofinetide approved in US, Canada and Israel, but CHMP recommended refusal in Europe
• Neuren fully supports Acadia's plan to re-examine CHMP decision

Neuren Pharmaceuticals (ASX: NEU) has advised that its partner, Acadia Pharmaceuticals (Nasdaq: ACAD), plans to request re-examination of the opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) regarding the Marketing Authorization Application for trofinetide for the treatment of Rett syndrome in patients two years of age and older. The previously announced CHMP trend vote was confirmed in the formal vote, resulting in a recommendation to refuse approval. Trofinetide is approved in the United States, Canada and Israel, where it represents the first and only treatment approved for Rett syndrome. Acadia's CEO stated the company remains committed to working with EU regulators to explore next steps and bring the therapy to patients, as the treatment has demonstrated meaningful benefits for people living with Rett syndrome. Neuren's CEO expressed full support for Acadia's plan to re-examine the CHMP opinion, noting the unmet medical need in Europe remains substantial and urgent. While the pivotal LAVENDER clinical trial successfully met its co-primary and key secondary endpoints, the CHMP cited perceived deficits including the limited magnitude of the treatment effect, the study not capturing all core Rett syndrome symptoms, and the influence of patient discontinuations on the assessment of longer-term outcomes.

Neuren fully supports Acadia's plan to request re-examination of the CHMP opinion on trofinetide for Rett syndrome, as the unmet medical need in Europe remains substantial and urgent.