Kazia Therapeutics gains US FDA Rare Paediatric Disease Designation for paxalisib in rare form of childhood brain cancer

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Kazia 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).

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