Acadia To Seek Re-Examination After CHMP Rejects Trofinetide For Rett Syndrome Marketing Application In EU

Acadia Pharmaceuticals Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the company’s Marketing Authorization Application for trofinetide for the treatment of Rett syndrome in patients aged two years and older. Acadia stated it plans to request a formal re-examination of the decision after reviewing the CHMP’s grounds for refusal.

“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,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “The strong engagement and positive feedback we have seen from patients, caregivers, and clinicians in the Rett community reinforce our belief in the treatment’s clinical value. We remain committed to working constructively with EU regulators to explore next steps and to bring this therapy to patients.”

Although the pivotal LAVENDER™ trial met its co-primary and key secondary endpoints, the CHMP cited several concerns. These included the view that the treatment effect observed at 12 weeks, while measurable, was limited in magnitude. The committee also noted that the study did not fully capture all core symptoms of Rett syndrome and raised questions about the interpretation of longer-term outcomes due to patient discontinuations over time.

“Our family and others who play an important role in the delivery of care know first-hand the challenges that individuals living with Rett syndrome face every day,” said Markus Schulze, caregiver and member of the Rett Syndrome Society Nordrhein-Westfalen from Germany. “It is our hope that this important therapy will be approved to help the EU Rett community better navigate life with Rett syndrome.”

Acadia said the CHMP’s feedback will inform its re-examination strategy as it seeks to address the committee’s concerns. The company continues to support trofinetide’s clinical value based on the trial results and real-world experience in other markets.Trofinetide is already approved in the United States, Canada, and Israel, where it is the first and only authorized treatment specifically indicated for Rett syndrome.