The treatment includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.
Kazia chief executive officer Dr James Garner said: “This award of ODD concludes a program of regulatory optimisation that Kazia has initiated for paxalisib over the past six months.
“As we orient paxalisib towards commercialisation, these special designations from FDA will allow us to move forward in the swiftest and most effective way possible.”
Shares have been more than 15% higher in early trade to A$1.13 and have risen from A$0.515 at the market close on August 4.
Orphan Drug Designation status
ODD is a special status accorded to drugs which are considered promising potential treatments for rare (orphan) diseases, generally defined as those which affect less than 200,000 cases per annum in the United States.
It can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), extending the effective life of a commercial product.
ODD status also provides opportunities for orphan grant funding by the FDA, as well as protocol assistance, and financial benefits, such as a waiver of new drug application fees under the Prescription Drug User Fees Act (PDUFA fees), which are just under US$3 million in FY2020.
Rare paediatric disease designation
The receipt of ODD follows award of Rare Paediatric Disease Designation (RPDD) for DIPG on August 7, 2020.
Dr Garner said: “Taken together, RPDD and ODD provide a powerful suite of incentives, opportunities and protections for the development of paxalisib in DIPG.
“We look forward to seeing initial data from the ongoing phase I study in DIPG at St Jude Children’s Research Hospital during the second half of calendar 2020.
“In parallel, we are working closely with collaborators, advisors, and researchers to determine the best path forward for paxalisib in this devastating disease.”
Summary of Paxalisib regulatory status to date.
Kazia expects to present further data from its ongoing phase II study of paxalisib in glioblastoma at the Society for Neuro-Oncology (SNO) Annual Meeting in November 2020.
Precise timing remains uncertain due to pandemic-related disruption in conference schedules, but the company expects to provide an update to investors at the earliest opportunity.
Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in the second half of 2020.