Posted by Michael Wonder on 07 Aug 2020
US FDA grants rare paediatric disease designation to paxalisib for DIPG
7 August 2020 - Kazia Therapeutics is pleased to announce that the United States FDA has awarded rare paediatric disease designation to Kazia’s paxalisib (formerly GDC-0084) for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly-aggressive childhood brain cancer.
Rare paediatric disease designation has been awarded following positive emerging preclinical data in DIPG, and with initial clinical efficacy data expected in 2H CY2020; positive clinical data may substantially enhance likelihood of a potential future priority review voucher.
