Neuren Pharmaceuticals (ASX:NEU) has received a Rare Paediatric Disease Designation from the US FDA for NNZ-2591 in Phelan-McDermid syndrome.
The designation can qualify the company for a priority review voucher (PRV) that can be redeemed for a priority review for a different product or sold to another sponsor.
Neuren is preparing for an end-of-phase 2 Meeting with the FDA in the third quarter of 2024 to discuss the development program for NNZ-2591 in Phelan-McDermid syndrome. Neuren previously reported results from a Phase 2 trial in which significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures.
The US FDA has approved Neuren’s DAYBUE (trofinetide) for treating Rett syndrome in adult and paediatric patients aged two and over. Neuren has granted Acadia Pharmaceuticals an exclusive worldwide licence for the development and commercialisation of trofinetide.
NNZ-2591 is Neuren’s second drug candidate. It is in Phase 2 development for multiple neurodevelopmental disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid and Pitt-Hopkins syndromes.
