Kazia Therapeutics (ASX:KZA), an Australian oncology-focused biotechnology company, has announced that the US FDA has granted Fast Track Designation (FTD) to its paxalisib (formerly GDC-0084) for the treatment of glioblastoma.
Glioblastoma is the most common and aggressive form of primary brain cancer.
FTD is designed to expedite the development of pharmaceutical products with the potential to address unmet medical needs in serious or life-threatening conditions.
The designation provides Kazia with enhanced access to FDA, including opportunities for face-to-face meetings and written consultation throughout the remaining development of paxalisib. It also means paxalisib is eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) submission.
Kazia said it now plans to begin initial preparatory activities for NDA filing for paxalisib in 2021 (calendar year).
According to CEO Dr James Garner, “in awarding Fast Track Designation to paxalisib, FDA has recognised the drug’s potential to meaningfully improve outcomes for patients with glioblastoma. This is a very powerful acknowledgement.
"The opportunities that Fast Track Designation creates, as we move towards an NDA filing, are of great value and have the potential to substantially accelerate the commercialisation of paxalisib. In particular, the ‘rolling review’ process enables Kazia to complete and submit substantial sections of our NDA filing in advance, saving time and reducing risk for the product. We look forward to working closely with FDA as we move into the final stage of development for paxalisib.”
The company said the specific indication for which FTD has been approved is “for the treatment of patients with newly diagnosed glioblastoma with unmethylated O6-Methylguaninemethyltransferase (MGMT) promotor status who have completed initial radiation with concomitant temozolomide.”
"This language precisely reflects the patient population studied in the ongoing phase II study, and is the primary proposed population for the GBM AGILE pivotal study, and is the intended indication at commercial launch," said the company.
Kazia said it completed recruitment to its phase two clinical trial of paxalisib in newly diagnosed glioblastoma in February 2020. Interim clinical data was presented at the American Association of Cancer Research (AACR) Virtual Annual Meeting II in June 2020. Overall survival was calculated at 17.7 months, which compares to the historical figure of 12.7 months for temozolomide, the existing FDA-approved standard of care. Kazia expects to present further data from this study in late 2020 and to conclude the study in early 2021.