Highlights:
- Marketing Authorization Application (MAA) for trofinetide submitted to the European Medicines Agency (EMA) by Acadia Pharmaceuticals.
- Potential approval in Q1 2026 for the treatment of Rett syndrome in adults and pediatric patients aged two years and older.
- Managed Access Programs expected to begin in Q2 2025 across Europe.
A Milestone for Rett Syndrome Treatment
Neuren Pharmaceuticals (ASX: NEU) announced that its partner, Acadia Pharmaceuticals (NASDAQ: ACAD), has submitted an MAA for trofinetide, aiming to make it the first and only approved therapy for Rett syndrome in the European Union.
Trofinetide is already marketed as DAYBUE™ in the United States and Canada, where it has been approved for Rett syndrome.
Neuren CEO Jon Pilcher stated:
“We are pleased to see the achievement of this very important milestone towards potentially bringing trofinetide to the Rett community in Europe.”
Key Financial Milestones for Neuren
Under its licensing agreement with Acadia, Neuren is eligible for significant milestone payments and royalties:
- Commercial Milestones:
- US$35 million upon first commercial sale in Europe.
- Additional payments for milestones in Japan and other regions.
- Sales Milestones:
- Up to US$363 million tied to escalating annual net sales.
- Royalties:
- Tiered royalties on net sales ranging from mid-teens to low twenties percent.
About Trofinetide and Neuren
Trofinetide is designed to address Rett syndrome, a severe neurodevelopmental disorder with no current treatment options in Europe. Its approval in the U.S. and Canada marked a major step forward for patients and caregivers.
Neuren is also advancing its second candidate, NNZ-2591, which is in Phase 2 trials for several neurodevelopmental disorders, including Phelan-McDermid syndrome, Pitt Hopkins syndrome, and Angelman syndrome, with promising early results.
Looking Ahead
- Potential EMA Approval: Q1 2026
- Managed Access Programs: Expected in Q2 2025
- First Commercial Sale Milestone Payments: Anticipated post-approval
This submission represents a significant step in addressing the unmet needs of patients with Rett syndrome in Europe while unlocking substantial commercial opportunities for Neuren and Acadia.
