The United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Kazia Therapeutics’ (NASDAQ:KZIA) Paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.
Paxalisib is being investigated in a phase II clinical trial of multiple drug therapies, under the sponsorship of the Pacific Pediatric Neuro-Oncology Consortium (PNOC).
Kazia CEO, Dr James Garner, commented, "childhood brain cancer has emerged as an important area of focus for the paxalisib program. We have been working for some years with several world-leading researchers in DIPG, one of the most aggressive childhood cancers.
The company said that a phase I study is nearing completion, evaluating paxalisib in diffuse intrinsic pontine glioma (DIPG), and final data submission for publication is anticipated by the end of calendar year 2022.
Kazia is scheduled participate in a webinar to discuss its activities in childhood brain cancer on 22nd June 2022.
FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
Shares of Kazia are trading higher 19 percent at $6.27 in the pre-market session.
