Kazia Therapeutics Ltd (ASX:KZA, NASDAQ:KZIA) has obtained an orphan drug designation (ODD) from the United States Food and Drug Administration (FDA) for the treatment of atypical rhabdoid / teratoid tumours (AT/RT), a rare and highly aggressive childhood brain cancer, with paxalisib.
An ODD is a special status given to promising potential treatments addressing very rare (orphan) diseases, generally defined to affect less than 200,000 cases per annum in the US.
The designation provides developers with up to seven years orphan drug exclusivity, offering protection for the company’s data which could be used to develop generic versions of paxalisib, effectively extending the life of a commercial product.
It also provides opportunities for grant funding, protocol assistance and tax credits, wherein the FDA will waive fees relating to regulatory filing in AT/RT, with potential savings of some A$4.26 million (US$3 million) if Kazia pursues approval in this indication.
“Important new opportunity for paxalisib”
“Childhood brain cancer has emerged as an important area of focus for the paxalisib program,” Kazia CEO Dr James Garner said.
“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 was previous awarded an ODD for the use of paxalisib 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 2 study led by the Pacific Pediatric Neuro-Oncology Consortium.
Initial data from the Phase 2 study is expected to be available in 2023, while final results from a Phase 1 study of paxalisib in DIPG, led by St Jude Children’s Research Hospital in Memphis, is expected by the end of this year.