The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) concluded its June 2026 meeting with a series of significant regulatory decisions, including a rare reversal of a previous negative opinion for Acadia Pharmaceuticals' trofinetide (Daybu) in Rett syndrome, a recommendation to revoke the marketing authorization of Amgen's avacopan (Tavneos) following data integrity concerns, six positive opinions for new medicines, and three application refusals. Among the most commercially significant outcomes was the committee's decision not to recommend a cardiovascular label expansion for Eli Lilly's tirzepatide (Mounjaro).
Trofinetide wins re-examination in Rett syndrome
The CHMP reversed its earlier negative opinion on Acadia Pharmaceuticals' trofinetide (Daybu), recommending marketing authorization for the treatment of neurobehavioral symptoms of Rett syndrome in adults and children aged five years and older. The revised indication is narrower than the original application, suggesting the committee concluded the benefit-risk balance was favorable only within a more clearly defined patient population.
The clinical evidence supporting the application remains unchanged from the original review, although the EMA has not disclosed the specific issues resolved during re-examination. A separate question-and-answer document has been published explaining the committee's rationale.
If confirmed by the European Commission, the decision would establish the first approved pharmacological therapy for Rett syndrome in the EU. Trofinetide has been marketed in the US as Daybue since 2023, and EU authorization would extend Acadia's commercial footprint into another orphan disease market while benefiting from the region's orphan drug exclusivity framework.
Avacopan faces EU market withdrawal
The committee also recommended revoking the marketing authorization for Amgen's Tavneos (avacopan) following completion of an Article 31 referral. Unlike most post-marketing regulatory actions, the recommendation was driven by concerns over the integrity of data supporting the original approval rather than by new evidence of reduced efficacy or emerging safety risks. Of note the US FDA has indicated it is planning to withdraw Taveneos's market approval in April this year.
Avacopan was approved for adults with severe active granulomatosis with polyangiitis and microscopic polyangiitis on the basis of the Phase III ADVOCATE trial. The CHMP concluded that newly identified data integrity issues undermine confidence in the trial sufficiently to alter the medicine's benefit-risk assessment.
If confirmed by the European Commission, the decision would remove the first complement C5a receptor inhibitor from the EU market. The recommendation does not affect the product's US regulatory status.
Tirzepatide falls short of cardiovascular label expansion
The CHMP declined to recommend a new indication for Eli Lilly's Mounjaro (tirzepatide) to reduce the risk of major cardiovascular events in adults with type 2 diabetes and established cardiovascular disease. The committee did, however, agree that the submitted cardiovascular outcomes data should be incorporated into the product information, allowing clinicians access to the evidence without granting a formal therapeutic claim.
Although the decision limits Lilly's near-term European label expansion, inclusion of the cardiovascular data in the Summary of Product Characteristics (SmPC) may still influence prescribing decisions where clinically appropriate.