Kazia Therapeutics awarded US FDA Orphan Drug Designation to paxalisib treatment for malignant glioma

Kazia Therapeutics Ltd's (ASX:KZA) (NASDAQ:KZIA) paxalisib treatment for malignant glioma has been granted Orphan Drug Designation (ODD) by the United States Food and Drug Administration (FDA).

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.