Kazia Therapeutics Shares Up After FDA Awards Orphan Drug Designation to Drug for A Rare Form Of Childhood Brain Cancer

Jun 17, 2022 By MarketDepth

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Kazia Therapeutics Limited (NASDAQ: KZIA) shares up about 15% on Friday after the Company announced that the United States Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Kazia’s paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.

“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. Recent data presented at the AACR conference by Dr Jeffery Rubens and colleagues from Johns Hopkins Medical School has shown the potential of the drug to also add benefit in AT/RT, another form of childhood brain cancer that is very poorly served by existing treatments. This represents an important new opportunity for paxalisib, and one that we continue to explore enthusiastically with our collaborators and advisors.”

Kazia CEO, Dr James Garner

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