Neuren’s partner Acadia secures positive CHMP opinion for DAYBU® in Rett syndrome, paving the way for first EU treatment approval and triggering significant milestone payments for Neuren.
- CHMP issues positive opinion for DAYBU® in Rett syndrome
- Potential first EU-approved treatment for Rett syndrome symptoms
- Neuren to receive US$35 million on first EU sale plus royalties
- European Commission decision expected in coming months
- DAYBU® already approved in US, Canada, and Israel
CHMP Endorses DAYBU® for Rett Syndrome in EU
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorisation for Neuren Pharmaceuticals’ licensed drug DAYBU® (trofinetide) to treat neurobehavioral symptoms of Rett syndrome in patients aged five and above. This positive opinion follows a re-examination procedure and, if ratified by the European Commission, would make DAYBU® the first approved therapy for this indication across the 27 EU member states plus Iceland, Liechtenstein, and Norway.
Financial Upside for Neuren on EU Launch
Under its exclusive worldwide licence agreement with Acadia Pharmaceuticals, Neuren stands to gain a US$35 million payment upon DAYBU®’s first commercial sale in Europe. Additional milestone payments could total up to US$170 million tied to escalating sales thresholds, alongside tiered royalties ranging from mid-teens to low-20s percentage of net sales. This potential revenue stream complements Neuren’s growing royalty income from DAYBU® in other markets, where the drug is already approved and generating sales.
Rett Syndrome: A Rare and Complex Disorder
Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, with an incidence of approximately one in 10,000 to 15,000 births. Characterised by early normal development followed by regression in communication and motor skills, the condition leads to lifelong disability requiring intensive care. The disorder stems from mutations in the MECP2 gene, impairing synaptic communication and brain plasticity. Until now, no approved treatment existed in the EU for the neurobehavioral symptoms that DAYBU® targets.
Next Steps and Market Implications
The European Commission is expected to issue its final decision on the marketing authorisation in the coming months. Approval would open a new market for Acadia’s commercialisation efforts and trigger substantial payments to Neuren. This milestone follows a period of regulatory challenges, including a prior refusal that Acadia successfully appealed through the CHMP re-examination process. The recommendation aligns with Neuren’s expanding footprint for DAYBU®, which has seen rising royalties and new formulations launched recently in other regions. The approval could also bolster confidence in Neuren’s pipeline, including its Phase 3 trials for NNZ-2591 in other neurodevelopmental disorders.
Bottom Line?
The CHMP’s positive opinion marks a pivotal regulatory milestone for Neuren, but the ultimate European Commission decision and subsequent market uptake will determine the financial impact.
Questions in the middle?
- Will the European Commission approve DAYBU® without further delay or conditions?
- How quickly can Acadia commercialise DAYBU® across the EU following approval?
- What impact might EU approval have on Neuren’s broader development pipeline and partnerships?