Acadia Pharmaceuticals is confronting a regulatory obstacle in Europe after the European Medicines Agency’s Committee for Human Medicinal Products (CHMP) issued a "negative trend vote" on the company’s Marketing Authorization Application (MAA) for trofinetide, its candidate therapy for Rett syndrome.
The stock was trading at $26.13 at the time of the notice, up from a prior close of $25.13. The CHMP action signals that, in its current form, the data package submitted by Acadia has not met the agency’s benefit-risk threshold for approval, a characterization echoed by H.C. Wainwright.
Following the CHMP vote, H.C. Wainwright reiterated a Buy rating on Acadia shares and maintained a price target of $37.00. That target falls within the broader analyst consensus, which continues to carry Buy opinions and price targets in a range from $17 to $40 per share.
With the CHMP’s negative trend vote, the process in Europe is expected to shift from a standard review to a re-examination. That procedural change is likely to introduce a delay, with a minimum additional wait time estimated at four to six months before a potential European approval decision could be reached.
Trofinetide is Acadia’s investigational treatment for Rett syndrome, a rare genetic neurological disorder that primarily affects females and produces serious impairments in brain function. The drug has already received regulatory approval in the United States, Canada, and Israel.
Acadia has indicated it plans to request a re-examination of the CHMP opinion on the trofinetide application, moving forward with the formal next step in the European review process.
Separately, the company provided forward-looking sales expectations for its portfolio. Acadia anticipates combined net sales of approximately $1.7 billion by 2028 for its approved and late-stage products: about $1 billion from NUPLAZID, its Parkinson’s disease psychosis therapy, and roughly $700 million from DAYBUE, the commercial name for trofinetide in Rett syndrome.
Market analysts retained a generally constructive view on the company despite the European setback. Canaccord Genuity reiterated a Buy rating with a $32.00 price objective, describing Acadia as undervalued on the basis of its approved products. Citizens likewise maintained a Market Outperform rating and a $34.00 price target in the wake of the CHMP action.
Looking ahead, Acadia is planning a phased commercial introduction of a new powder formulation of trofinetide, DAYBUE STIX. The company intends a limited launch in early 2026 followed by a full market roll-out later that year. The new formulation is positioned to provide a dye- and preservative-free option for patients with Rett syndrome.
Sectors impacted: Biotechnology, pharmaceuticals and equity markets.