Kazia Shares Shoot Higher As FDA Grants Rare Pediatric Disease Designation To Brain Cancer Candidate

The United States Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation (RPDD) to Kazia Therapeutics’ KZIA lead drug Paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT).

AT/RT is a rare and highly-aggressive childhood brain cancer, most common cause of cancer death in children, and outcomes in many forms of childhood brain cancer have not improved in decades.

Dr James Garner, CEO, commented, "this is the second time that paxalisib has been granted RPDD, and it demonstrates the importance of childhood brain cancer in the overall paxalisib development program. We very much hope that paxalisib can make a difference to families affected by both DIPG and AT/RT, and we will be working closely with clinicians, researchers, and FDA to determine the optimal way to move the drug forward.”

Paxalisib is currently investigated in a Phase 1 study for patients with Diffuse intrinsic pontine glioma (DIPG), final data readout from the trial is expected to be submitted for publication by the end of calendar year 2022.

Recently, Paxalisib was granted orphan drug designation (ODD) for AT/RT by the regulatory authority.

Rare Pediatric Disease Designation (RPDD) is granted to drugs which are under development for rare childhood diseases.

Price Action: Kazia shares are trading about 13 percent high at $4.72 on Wednesday's pre-market session at the time of publication.