Neuren Pharmaceuticals (ASX:NEU) announced that its North American partner, Acadia Pharmaceuticals (NASDAQ:ACAD), will formally request a re-examination of its marketing application for trofinetide in Europe.
The move follows a negative opinion issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use, which was formally adopted this week after an initial "negative trend vote" in February.
Despite the drug being the first approved treatment for Rett syndrome in the United States, Canada, and Israel, the CHMP cited concerns regarding the clinical trial data.
The committee noted that the magnitude of the treatment's effect over 12 weeks appeared limited and suggested the study failed to capture all core symptoms of the rare neurodevelopmental disorder.
Neuren CEO Jon Pilcher expressed strong support for the appeal, highlighting the drug's established track record.
"Trofinetide has been making a difference for patients for nearly three years in approved markets, and the unmet medical need in Europe remains substantial and urgent," Pilcher noted.
A final decision is now anticipated by the end of the second quarter of 2026.
At the time of reporting, Neuren Pharmaceuticals’ share price was $12.66.