Australian oncology-focused biotechnology company Kazia Therapeutics (ASX: KZA) has been granted Fast Track Designation by the US Food & Drug Administration for its lead drug paxalisib (formerly GDC-0084) for the treatment of glioblastoma.
FTD is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions such as glioblastoma, which currently ranks as the most common and most aggressive form of primary brain cancer.
It provides Kazia with enhanced access to the USFDA, including opportunities for face-to-face meetings and written consultations throughout the remaining term of paxalisib’s development.
The specific indication governing the designation is stated as being “for the treatment of patients with newly-diagnosed glioblastoma with unmethylated O6-Methylguaninemethyltransferase (MGMT) promotor status who have completed initial radiation with concomitant temozolomide”.
The indication reflects the patient population being studied in Kazia’s ongoing Phase II study into paxalisib, and is the primary proposed population for the international, academic-led, multi-drug GBM AGILE trial.
Kazia was invited to include paxalisib in the trial earlier this year and recruitment is expected to begin before year end.
Kazia chief executive officer Dr James Garner said the designation recognises the important role paxalisib could play in the treatment of brain cancers.
“It is a very powerful acknowledgement by the USFDA which recognises [our] drug’s potential to meaningfully improve outcomes for patients with glioblastoma,” he said.
“The opportunities this designation creates, as we move towards the submission of a new drug application are of great value and have the potential to substantially accelerate the commercialisation of paxalisib.”
He said a notable benefit would be the ‘rolling review’ process which enables Kazia to complete and submit substantial sections of its new drug application in advance, saving time and reducing product risk.
Introduced under the USFDA Modernisation Act (1997), FTD is designed to allow patients to access new drugs as quickly as possible.
The designation must be requested by the sponsor company and accompanied by a detailed review of preclinical and clinical data.
To be awarded FTD, a drug must generally be able to show some potential advantage over existing therapies, either in terms of safety or efficacy.
Paxalisib is now eligible for Accelerated Approval (in which a new medicine is approved prior to the availability of definitive data) and Priority Review (which reduces the standard 12-month review process to six).