Neuren Pharmaceuticals Ltd (ASX: NEU) shares are under pressure on Tuesday.
In morning trade, the ASX 200 stock is down over 6% to $12.89.
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Why is this ASX 200 stock under pressure?
This morning, Neuren advised that its partner, Acadia Pharmaceuticals (NASDAQ: ACAD), has confirmed that it plans to request re-examination of the opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
This is in relation to the marketing authorisation application for trofinetide for the treatment of Rett syndrome in patients two years of age and older.
Neuren also confirmed that, as was anticipated, the previously announced CHMP trend vote was confirmed in a formal vote, blocking its immediate approval.
Trofinetide is approved in the United States, Canada and Israel, where it represents the first and only treatment approved for Rett syndrome.
Acadia's CEO, Catherine Owen Adams, commented:
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 strong engagement and positive feedback we have seen from patients, caregivers, and clinicians in the Rett community reinforce our belief in the treatment's clinical value. We remain committed to working constructively with EU regulators to explore next steps and to bring this therapy to patients.
Why was approval denied?
The ASX 200 stock highlights that while the pivotal LAVENDER clinical trial successfully met its co-primary and key secondary endpoints, the CHMP issued a refusal based on perceived deficits.
This includes the treatment effect observed with trofinetide after 12 weeks, while measurable, was viewed as limited in magnitude. It also notes that the study did not capture all core symptoms of Rett syndrome and that assessment of longer‑term outcomes was influenced by patient discontinuations over time.
Acadia believes this feedback provides important information as it considers the intended re-examination.
Commenting on the news, the ASX 200 stock's CEO, Jon Pilcher, said:
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.
Following today's weakness, the Neuren share price is now down by a disappointing 30% since the start of the year. However, it is trading relatively flat on an annual basis.
