Acadia Pharmaceuticals plans to re-examine Rett syndrome treatment following EU setback

The news: Neuren Pharmaceuticals announced that its US listed partner, Acadia Pharmaceuticals, will seek a re-examination of the European market authorisation application for Rett syndrome treatment, trofinetide following a setback from European Medicines Agency (EMA) committee.

The context: This follows a “negative trend vote” earlier in February for trofinetide from the EMA, despite already being approved in the US, Canada and Israel for the treatment of Rett syndrome.

The Committee for Medicinal Products for Human Use (CHMP) issued a refusal for market commercialisation in Europe, citing concerns over perceived deficits within the clinical trial, including limited in magnitude of effect and failure to capture all core symptoms of Rett syndrome.

Arcadia said the feedback provides crucial information for the intended re-examination process.

What they said: "Neuren fully supports a re-examination of the CHMP opinion. 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,” Neuren Pharmaceuticals CEO Jon Pilcher said.