New orphan drug designation for Kazia Therapeutics' paxalisib

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Kazia Therapeutics (ASX:KZA) has announced that the US FDA has granted Orphan Drug Designation (ODD) to its paxalisib for the treatment of atypical rhabdoid/teratoid tumours (AT/RT).

These tumours are a rare and highly aggressive childhood brain cancer.

CEO Dr James Garner said, “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.”

ODD provides an additional period of 7.5 years of data exclusivity (for a paediatric disease). It also means the FDA will waive fees for a marketing application.

Kazia previously received ODD for paxalisib in glioblastoma and malignant glioma.