Can Neuren Overcome the Latest EU Setback for Its Rett Syndrome Treatment?

Source: Kapitales Research

Highlights

• Neuren Pharmaceuticals Limited (ASX: NEU) has provided an update following a regulatory setback for trofinetide in Europe.
• At the time of writing, European regulators have issued a negative trend vote during the initial marketing authorisation review.
• The company’s partner plans to request a formal re-examination, keeping the European approval pathway active.

Regulatory Update in Europe

Neuren Pharmaceuticals Limited (ASX: NEU) has informed the market that its global licensing partner, Acadia Pharmaceuticals, has received feedback from European regulators regarding trofinetide, a treatment for Rett syndrome. At the time of writing, the Committee for Medicinal Products for Human Use has indicated an unfavourable preliminary outcome following its review of the European marketing authorisation application.

While this outcome was unexpected, the European regulatory framework allows applicants to seek a re-examination once a formal opinion is delivered. Acadia has indicated that it intends to pursue this option, subject to the final opinion expected in February. This process allows for further engagement with regulators and the opportunity to present additional supporting data and analysis.

Strength of Existing Clinical and Real-World Evidence

Despite the setback in Europe, trofinetide has already received regulatory approval in several key markets. At the time of writing, the therapy is approved for use in the United States, Canada, and Israel, with more than 1,000 patients currently receiving treatment worldwide. Ongoing real-world evidence from the United States continues to align with outcomes observed in clinical trials, supporting confidence in the treatment’s effectiveness.

The available data spans a broad patient population, ranging from young children to adults, reinforcing the robustness of the existing safety and efficacy profile.

Neuren’s Position and Broader Pipeline

Neuren’s management has acknowledged disappointment with the European feedback but has reiterated its commitment to pursuing the re-examination process. At the time of writing, the company remains focused on expanding access to therapies for patients with significant unmet medical needs.

Beyond trofinetide, Neuren continues to progress its second drug candidate, NNZ-2591, which is being developed across multiple rare neurodevelopmental disorders. Phase 2 trials have produced encouraging results, and the program has received orphan drug designation in both the United States and Europe.

Outlook

At the time of writing, investor focus is likely to remain on the outcome of the European re-examination process for trofinetide. While regulatory uncertainty remains in the near term, existing approvals and growing real-world use provide a solid foundation. Progress across Neuren’s broader development pipeline may also increasingly influence sentiment as discussions with European regulators continue.

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