FDA Awards Rare Pediatric Disease Designation To Kazia's Paxalisib For Childhood Brain Cancer

(RTTNews) - The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to Kazia Therapeutics Ltd's (KZIA, KZA.AX) paxalisib for the treatment of atypical rhabdoid / teratoid tumors, a rare and highly-aggressive childhood brain cancer, the company said in a statement.

In Wednesday pre-market trade, KZIA was trading at $4.76 up $0.59 or 14.15 percent.

Rare Pediatric Disease Designation means that the sponsor may be entitled to receive a pediatric priority review voucher (pPRV) if the drug is initially approved for that rare childhood disease. A priority review voucher grants the holder an expedited six-month review of a new drug application.

Paxalisib was previously granted orphan drug designation for atypical rhabdoid / teratoid tumors by FDA on 16 June 2022.

The companay noted that a phase II clinical trial of multiple drug therapies, including paxalisib, is ongoing, under the sponsorship of the Pacific Pediatric Neuro-Oncology Consortium. The study combines several investigational drugs in the treatment of patients with diffuse midline gliomas (DMGs), a category which includes DIPG. Initial data from the study is anticipated in 2023.

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