Kazia Therapeutics, an Australian biotech company, has secured the rare pediatric disease designation (RPDD) for paxalisib (formerly GDC-0084) from the US Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG).
Diffuse intrinsic pontine glioma is considered to be a rare and highly-aggressive childhood brain cancer.
The rare pediatric disease designation for paxalisib based on positive emerging preclinical data in diffuse intrinsic pontine glioma and with initial clinical efficacy data likely to be released in 2H CY2020.
According to Kazia Therapeutics, positive clinical data could considerably increase the chances of a potential rare pediatric disease priority review voucher in the future.
Paxalisib is a small molecule inhibitor of the PI3K / AKT / mTOR pathway. It is being developed for the treatment of glioblastoma, the most usual and most aggressive type of primary brain cancer in adults.
Licensed in late 2016 from Genentech, paxalisib advanced into a phase 2 clinical trial in 2018. Paxalisib was granted orphan designation from the US FDA for glioblastoma in 2018.