Acadia provides update on EU regulatory submission
| Stock | Neuren Pharmaceuticals Ltd (NEU.ASX) |
|---|---|
| Release Time | 3 Feb 2026, 9:40 a.m. |
| Price Sensitive | Yes |
Acadia provides update on EU regulatory submission for trofinetide
- Acadia received a negative trend vote from the CHMP of the EMA on its marketing authorization application for trofinetide for Rett syndrome treatment in the EU
- Acadia intends to request a re-examination of the CHMP opinion if formally adopted
- Neuren CEO expresses frustration with the negative trend vote given the positive clinical trial and real-world experience with trofinetide
Neuren Pharmaceuticals (ASX: NEU) announced that its licensee Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) has provided an update on its marketing authorization application (MAA) for trofinetide for the treatment of Rett syndrome in the European Union (EU). Acadia was informed by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) of a negative trend vote on its MAA for trofinetide for the treatment of Rett syndrome, following its recent CHMP oral explanation. Subject to the outcome of the CHMP vote in February, Acadia intends to request a re-examination of the opinion by the CHMP upon its formal adoption. Acadia's CEO, Catherine Owen Adams, stated that while the negative trend vote is disappointing, the strong data that supported the approval of trofinetide in the United States, Canada, and Israel demonstrate the meaningful benefits that trofinetide can deliver. Acadia has more than 1,000 patients on active treatment globally, and its ongoing real-world experience study in the U.S. continues to show outcomes that closely mirror the impact observed in rigorous randomized clinical trials conducted across a broad age range. Acadia remains committed to advancing trofinetide as an important potential treatment option in the EU and is dedicated to making it available to individuals and families who urgently need a new therapeutic option. Neuren's CEO, Jon Pilcher, expressed frustration with the negative trend vote, given the totality of experience with trofinetide in clinical trials and real-world use over many years, and fully supports Acadia's intention to seek re-examination of the CHMP opinion in February, if necessary.