Acadia Pharmaceuticals announced that it will request a re‑examination of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) negative opinion on its Rett syndrome therapy trofinetide.
The CHMP adopted a negative opinion on March 2, 2026, citing limited treatment effect after 12 weeks, incomplete capture of core Rett symptoms, and concerns about long‑term outcomes due to patient discontinuations.
Acadia argues that the pivotal LAVENDER trial met its co‑primary and key secondary endpoints, demonstrating meaningful benefits for patients and caregivers. The company says the trial’s data support a more favorable benefit‑risk profile than the CHMP’s assessment.
Trofinetide, marketed as DAYBUE, is already approved in the United States (March 2023), Canada, and Israel, where it is the first and only treatment for Rett syndrome. The EU market represents a substantial unmet need, and the company believes the therapy could fill that gap if approval is secured.
Acadia’s Q4 2025 results showed GAAP total revenue of $284 million and net income of $274 million, with earnings per share of $1.60, a significant beat over the $0.14 consensus. Full‑year 2025 revenue reached $1.07 billion and net income $391 million, EPS $2.30, underscoring the company’s strong financial position. Management remains confident, with CEO Catherine Owen Adams stating, "While we are disappointed by the CHMP's recommendation to refuse approval, we continue to be encouraged by the meaningful benefits trofinetide has demonstrated for people living with Rett syndrome…" The company also highlighted that it will continue to engage constructively with EU regulators to explore next steps.
Neuren Pharmaceuticals, Acadia’s partner, also expressed support: "Neuren fully supports a re‑examination of the CHMP opinion. Trofinetide has been making a difference for patients for nearly three years in approved markets and the unmet medical need in Europe remains substantial and urgent."
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