The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) informed Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) of a negative vote on its Marketing Authorization Application (MAA) for trofinetide for 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.
"While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver," said Catherine Owen Adams, Chief Executive Officer.
What Next?
Pursuant to EU legislation, an applicant has the right to request a re-examination of a CHMP opinion within 15 calendar days of receipt of the opinion, followed by submission of the grounds for the request for re-examination within 60 calendar days of receipt of the opinion.
The CHMP has up to 60 days after receipt of these grounds to re-examine its opinion.
In March 2023, the FDA approved Acadia's Daybue (trofinetide) for Rett syndrome in adult and pediatric patients two years of age and older.
Daybue is the first and only drug approved for treating Rett syndrome, a rare genetic neurological and developmental disorder that causes a progressive loss of motor skills and language. Rett syndrome primarily affects females.
In September 2025, Acadia Pharmaceuticals released topline results on Wednesday from its Phase 3 COMPASS PWS trial evaluating the efficacy and safety of intranasal carbetocin (ACP-101) in patients with hyperphagia in Prader-Willi syndrome (PWS).
Intranasal carbetocin did not demonstrate a statistically significant improvement over placebo on the study's primary endpoint, change from baseline to Week 12 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), nor was there separation from placebo on any secondary endpoint.
ACAD Price Action: ACADIA Pharmaceuticals shares were down 6.12% at $24.53 at the time of publication on Tuesday, according to Benzinga Pro data.
Photo: Piotr Swat via Shutterstock
