FDA Awards Orphan Drug Designation To Kazia's Paxalisib For Rare Form Of Childhood Brain Cancer

(RTTNews) - Kazia Therapeutics Limited (KZIA, KZA.AX) said that the U.S. Food and Drug Administration has awarded Orphan Drug Designation to the company's paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.

Orphan Drug Designation is a special status accorded to drugs which are considered promising potential treatments for rare diseases, generally defined as those which affect less than 200,000 cases per annum in the United States.

Paxalisib has previously been granted Orphan Drug Designation in malignant glioma, a category of brain cancer that includes both glioblastoma, an adult brain cancer which is the lead indication for the drug, and diffuse intrinsic pontine glioma (DIPG), a rare childhood brain cancer which is currently the subject of a phase II study led by the Pacific Pediatric Neuro-Oncology Consortium.