- The ADRs of Australian biotech Kazia Therapeutics ( NASDAQ: KZIA ) gained 8% Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) granted the Rare Pediatric Disease Designation for an experimental therapy targeted at a form of childhood cancer.
- Paxalisib a brain-penetrant inhibitor received the designation as a treatment for atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly aggressive childhood brain cancer, Kazia ( KZIA ) said in a press release.
- In awarding the RPD tag, the federal agency picks drug candidates targeted at serious and life-threatening diseases affecting less than 200K individuals in the U.S. with a primary impact on children aged 18 years or younger.
- If a drug with a rare pediatric disease designation wins FDA approval, the sponsor of its marketing application is entitled to receive a Priority Review Voucher (PRV) from the regulator.
- The PRVs valued at more than $100M historically, can be redeemed to receive a priority review for any subsequent marketing application, or it can be sold.
- In June, Kazia ( KZIA ) announced that the FDA awarded orphan drug designation for Paxalisib for the same condition.
For further details see:
Kazia gains on FDA’s rare pediatric disease designation for childhood cancer therapy