Kazia: FDA says data does not support accelerated approval for glioblastoma treatment

  Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, today provided a regulatory update on paxalisib for the treatment of glioblastoma (GBM) following its Type C clinical meeting with the United States Food and Drug Administration (FDA).

In July 2024, the Company reported topline results from the GBM-AGILE study in which newly diagnosed unmethylated (NDU) patients with glioblastoma treated with paxalisib showed a clinically meaningful improvement in a prespecified secondary analysis for overall survival (OS) compared to standard of care. Based on these results and the totality of data from all completed paxalisib clinical studies, Kazia requested a meeting with the FDA to discuss potential clinical and regulatory paths forward.

Following discussions with the FDA and feedback from Kazia's recent Type C meeting, the FDA's current position is that data on OS would generally not be appropriate for accelerated approval, but could be considered to support a traditional/standard approval. The Agency further commented that the secondary endpoint OS data from the GBM-AGILE study are supportive and informative for designing and executing a pivotal registrational study in pursuit of a standard approval.  Importantly, the Company aligned with the FDA on key aspects of the design of a proposed registrational/pivotal phase 3 study, including patient population, primary endpoint, and the comparator arm to be used.

https://www.prnewswire.com/news-releases/kazia-therapeutics-provides-update-on-paxalisib-regulatory-pathway-following-type-c-meeting-with-fda-302340490.html