EU Rejects Trofinetide for Rett Syndrome, Acadia Plans Re-Examination
CHMP adopted a negative opinion on Acadia's EU marketing application for trofinetide to treat Rett syndrome, citing modest 12-week effects and symptom coverage gaps. Acadia will seek a re-examination and faces a 7.9% share drop over six months despite targeting $1.7 billion in combined Nuplazid and Daybue sales by 2028.
1. CHMP Rejection Details
The CHMP formally adopted a negative opinion on Acadia’s marketing application for trofinetide in the EU, citing modest 12-week efficacy in the pivotal LAVENDER study, incomplete coverage of core Rett syndrome symptoms and concerns over long-term outcome data due to patient discontinuations.
2. Re-Examination Strategy
Following a detailed review of the CHMP’s grounds for refusal, Acadia plans to request an official re-examination, leveraging the trial insights to address efficacy magnitude, symptom assessment and data continuity in hopes of overturning the negative opinion.
3. EU Market Implications
The rejection delays trofinetide’s potential entry into a rare disease market affecting approximately one in 10,000-15,000 female births, deferring incremental EU revenue that could have supplemented the drug’s U.S. launch under the Daybue brand.
4. Financial Outlook and Share Performance
Despite this regulatory setback, Acadia projects combined net sales of $1.7 billion by 2028 from Nuplazid and Daybue, though its shares have fallen 7.9% over the past six months as investors weigh European approval uncertainty.