Kazia Therapeutics Ltd (ASX:KZA, NASDAQ:KZIA) has been awarded a Rare Paediatric Disease Designation (RPDD) by the US Food and Drug Administration (FDA) for the use of paxalisib in the treatment of atypical rhabdoid/teratoid tumours (AT/RT), a rare and highly aggressive childhood brain cancer.
The RPDD is granted to drugs with the potential to treat rare childhood diseases, providing a paediatric priority review voucher (pPRV) if the drug is initially approved for that rare childhood disease.
A pPRV allows an expedited six-month review process for a new drug application. As a tradable voucher, a pPRV can command prices of more than A$147 million (US$100 million).
Targeting brain cancer indications
“This is the second time that paxalisib has been granted RPDD and it demonstrates the importance of childhood brain cancer in the overall paxalisib development program,” Kazia Therapeutics CEO Dr James Garner said.
“Brain cancer is the most common cause of cancer death in children and outcomes in many forms of childhood brain cancer have not improved in decades.
“We very much hope that paxalisib can make a difference to families affected by both DIPG and AT/RT, and we will be working closely with clinicians, researchers and FDA to determine the optimal way to move the drug forward.”
The FDA awarded paxalisib the RPDD following a presentation of promising preclinical data for paxalisib in AT/RT, presented by Professor Jeffrey Rubens and colleagues at the American Association of Cancer Research (AACR) Annual Meeting in New Orleans, US, in April this year.
This latest RPDD is the second awarded to paxalisib, the first being for diffuse intrinsic pontine glioma (DIPG), a childhood brain cancer that affects the brainstem. About 300 children are diagnosed with DIPG in the US each year.