Acadia Pharmaceuticals reported that the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency signaled a negative trend vote on its Marketing Authorization Application for trofinetide, its treatment for Rett syndrome.
The negative signal follows an oral explanation meeting with the CHMP held on January 27. Acadia said it expects a final CHMP vote in February and intends to request a re-examination of the committee's opinion once the vote is formally adopted.
Trofinetide is marketed in the United States as Daybue and already holds regulatory approvals in Canada and Israel for the treatment of Rett syndrome, a rare neurological disorder. Acadia also markets Nuplazid, a therapy indicated for Parkinson's disease psychosis.
Shares of Acadia were trading at $26.13, reflecting a market capitalization of $4.42 billion. Financial data cited by the company shows a balance sheet with more cash than debt.
Analyst reactions
Despite the CHMP development, several research firms maintained positive ratings on Acadia's stock. Canaccord Genuity reiterated a Buy rating with a $32.00 price target, pointing to the real-world experience with trofinetide, the clinical data that supported FDA approval, and the potential to manage side effects over time.
Canaccord also highlighted Acadia's sales potential, estimating combined peak U.S. sales from Daybue and Nuplazid in the range of $1.5 billion to $2.0 billion. In another analyst projection cited by the company, Acadia expects combined sales of Nuplazid and Daybue of $1.7 billion by 2028.
Other firms maintained favorable views as well. H.C. Wainwright reiterated a Buy rating with a $37.00 target, while Citizens kept a Market Outperform rating with a $34.00 price target. All three firms preserved their positive assessments despite the CHMP's negative trend vote.
Context and next steps
The CHMP's negative trend vote is an interim signal preceding a formal committee decision. Acadia has publicly stated it will seek re-examination if the committee's final opinion, expected in February, is adopted in its current form. The company has not disclosed additional regulatory filings or amendments beyond the planned request for re-examination.
For now, trofinetide remains authorized in the U.S., Canada, and Israel, and Acadia continues to emphasize its broader commercial opportunity across Daybue and Nuplazid as well as additional programmes in its pipeline.