FDA Grants Rare Pediatric Disease Designation to Kazia for Childhood Brain Cancer Drug

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Kazia Therapeutics for its experimental therapy paxalisib for the treatment of a rare and highly aggressive childhood brain cancer known as diffuse intrinsic pontine glioma.

The FDA awarded the designation following positive emerging preclinical data in diffuse intrinsic pontine glioma (DIPG), and with initial clinical efficacy data expected in second half of 2020.

For patients diagnosed with DIPG, there are currently no FDA-approved drug treatments, and the average survival from diagnosis is around 9.5 months.

Paxalisib is a small molecule inhibitor of the PI3K / AKT / mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults. Licensed from Genentech in late 2016, paxalisib entered a phase 2 clinical trial in 2018. The FDA granted paxalisib orphan designation as well.

“Although glioblastoma remains our primary focus for paxalisib, we have been devoting increasing energy to developing the drug in childhood brain cancer as well,” said James Garner, CEO of Kazia. “The granting of RPDD by the FDA recognizes our efforts and achievements so far and leaves us well placed to move paxalisib forward as a potential therapy for DIPG.”

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 approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: James Garner, CEO of Kazia

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.