• Neuren receives Rare Pediatric Disease Designation from FDA for NNZ-2591 in Pitt Hopkins syndrome, Angelman syndrome, and Phelan-McDermid syndrome
• Designation may allow Neuren to receive a priority review voucher if NNZ-2591 receives marketing authorization for any of these indications
• Priority review voucher can be redeemed or sold to another sponsor

Neuren Pharmaceuticals (ASX: NEU) has received Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its drug candidate NNZ-2591 in each of Pitt Hopkins syndrome (PTHS), Angelman syndrome (AS), and Phelan-McDermid syndrome (PMS). This designation may allow Neuren to be awarded a priority review voucher (PRV) if the Rare Pediatric Disease PRV program is reauthorized by the US Congress and NNZ-2591 receives marketing authorization for any of these indications. The Rare Pediatric Disease PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor. As previously reported, Neuren's partner Acadia Pharmaceuticals received a PRV for DAYBUE™ (trofinetide) in Rett syndrome and sold the PRV for US$150 million. Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. All of Neuren's programs have been granted 'orphan drug' designation in the United States, which provides incentives to encourage development of therapies for rare and serious diseases.