FDA Awards Rare Pediatric Disease Designation to Kazia’s Paxalisib

The U.S. Food and Drug Administration has awarded Rare Pediatric Disease Designation to Kazia Therapeutics’ experimental therapy paxalisib for the treatment of atypical rhabdoid/teratoid tumors, a rare and aggressive childhood brain cancer.

Photo: James Garner, CEO of Kazia Therapeutics

Paxalisib, a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults. Kazia licensed paxalisib from Genentech in late 2016. The company began recruitment to GBM AGILE, a pivotal study in glioblastoma, in January 2021. Seven additional studies are active in various forms of brain cancer.

The FDA granted Orphan Drug designation for paxzlisib for glioblastoma in February 2018, and Fast Track designation for glioblastoma in August 2020. In addition, paxalisib was granted Rare Pediatric Disease designation and Orphan Drug designation for DIPG in August 2020, and for atypical rhabdoid/teratoid tumors in June 2022.

FDA’s award of Rare Pediatric Disease designation follows the presentation of promising preclinical data for paxalisib in atypical rhabdoid/teratoid tumors at the American Association of Cancer Research Annual Meeting in New Orleans, LA, in April 2022.

Paxalisib was previously granted orphan drug designation (ODD) for AT/RT by FDA on 16 June 2022.

“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,” said James Garner, CEO of Kazia. “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.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes paxalisib eligible for a Rare Pediatric Disease Priority Review voucher upon, which can be used to shorten the expected review time for a new drug application to six months from 10 months.

The vouchers are potentially lucrative because they can be sold. Most recently, BridgeBio Pharma sold a priority review voucher for $110 million.

Author: Rare Daily Staff