Neuren Pharmaceuticals (ASX:NEU) has received a Rare Paediatric Disease Designation from the US FDA for NNZ-2591 in Pitt-Hopkins syndrome and Angelman syndrome.
Neuren also received a Rare Pediatric Disease Designation from the FDA for NNZ-2591 in Phelan-McDermid syndrome.
The company said the designation means it may be awarded a priority review voucher if the Rare Paediatric Disease PRV program is reauthorised by the US Congress and NNZ-2591 receives marketing authorisation for any of the indications.
The Rare Paediatric Disease PRV program is designed to incentivise drug development for serious, rare paediatric diseases. If awarded, a voucher can be redeemed to receive a priority review for a different product or sold to another sponsor. Neuren's partner, Acadia Pharmaceuticals, received a PRV for DAYBUE (trofinetide) in Rett syndrome and sold it for US$150 million.
