Australian company Neuren Pharmaceuticals (ASX:NEU) has welcomed confirmation that the Committee for Medicinal Products for Human Use adopted a positive opinion recommending a European marketing authorisation for DAYBU (trofinetide) following a re-examination procedure.
The recommendation from the committee covers the treatment of neurobehavioral symptoms of Rett syndrome in adults and paediatric patients aged 5 years and older, and, if confirmed by the European Commission, would make DAYBU the first therapy approved for this indication in the European Union.
Investors welcomed the announcement by boosting the company's share price by over 25 per cent.
Neuren CEO Jon Pilcher said, “I am so delighted for all stakeholders to see this positive outcome from the CHMP re-examination process recommending marketing authorisation for DAYBU in the EU. With no approved treatment currently available in the EU, approval of DAYBU would represent an important step forward for patients, caregivers and the wider Rett syndrome community profoundly impacted by this devastating condition.”
Neuren requested the re-examination earlier this year after the committee's decision to refuse approval of DAYBU
The European Commission will review the CHMP opinion and is expected to issue a final decision in the coming months. A favourable EC decision would extend marketing authorisation across the 27 EU member states and also apply to Iceland, Liechtenstein, and Norway.
Under an exclusive licence granted to Acadia Pharmaceuticals, Neuren stands to receive US$35 million following the first commercial sale in Europe, as well as tiered royalties on net sales and potential milestone payments of up to US$170 million tied to escalating annual net sales thresholds.
Rett syndrome is a rare neurodevelopmental disorder affecting roughly one in every 10,000 to 15,000 female births worldwide. Symptoms typically include loss of acquired communication skills, purposeful hand use, hand stereotypies, and gait abnormalities, and most individuals require intensive round-the-clock care into adulthood.
DAYBU is already approved by the US Food and Drug Administration, Health Canada, and the Ministry of Health in Israel. The company is also advancing a second candidate, NNZ 2591, which has shown positive Phase 2 results across several neurodevelopmental disorders and is currently being evaluated in a Phase 3 trial in children with Phelan-McDermid syndrome.
