Neuren Pharmaceuticals Limited (ASX: NEU) has welcomed a significant development regarding its partnered drug, DAYBU® (trofinetide), for the European market. Neuren Pharmaceuticals develops new drug therapies to treat multiple serious neurological disorders caused by genetic abnormalities or brain injury, which currently have limited or no approved treatment options. The company’s partner, Acadia Pharmaceuticals Inc., announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion following a re-examination procedure, recommending European Union (EU) marketing authorisation for DAYBU®.
This recommendation is for DAYBU® (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and paediatric patients aged five years and older. If granted by the European Commission (EC), DAYBU® would become the first therapy approved for this indication in the EU, representing a critical advancement for patients. Following the CHMP recommendation, the EC is expected to issue a final decision in the coming months, which, if positive, would see the marketing authorisation apply across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. Neuren CEO Jon Pilcher expressed his delight, stating that with no approved treatment currently available in the EU, approval of DAYBU® would be an important step forward for patients, caregivers, and the wider Rett syndrome community.
Under the exclusive worldwide licence agreement with Acadia Pharmaceuticals Inc., Neuren stands to receive substantial financial benefits. Upon the first commercial sale of DAYBU® in the EU, Neuren is entitled to receive US$35 million. Additionally, the agreement includes potential sales milestone payments of up to US$170 million upon the achievement of escalating annual net sales thresholds, alongside tiered royalties ranging from mid-teens to low-20s per cent of net sales. This positive opinion from the CHMP underscores the potential for DAYBU® to address a significant unmet medical need and generate considerable returns for Neuren.
