By Elias Schisgall

A European regulatory body adopted a negative opinion regarding Acadia Pharmaceuticals' application for trofinetide, its treatment for Rett syndrome, a rare neurodevelopmental disorder seen in children.

The pharmaceutical company said Monday that it was disappointed by the recommendation and plans to request a re-examination of the decision.

The Committee for Medicinal Products for Human Use, part of the European Medicines Agency, said a pivotal trial for trofinetide showed only a limited treatment effect, adding that the company did not capture all the core symptoms of Rett syndrome and that patient discontinuations influenced the assessment of longer-term outcomes, Acadia said.

"While we are disappointed by the CHMP's recommendation to refuse approval, we continue to be encouraged by the meaningful benefits trofinetide has demonstrated for people living with Rett syndrome," Chief Executive Officer Catherine Owen Adams said. "We remain committed to working constructively with EU regulators to explore next steps and to bring this therapy to patients."

Trofinetide is approved for use in the U.S., Canada, and Israel, the company said.

Write to Elias Schisgall at elias.schisgall@wsj.com

(END) Dow Jones Newswires

March 02, 2026 17:03 ET (22:03 GMT)

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