FDA doles out two more rare pediatric designations

The US Food and Drug Administration over the last couple of day has issued two coveted Rare Pediatric Disease designations (RPDD).

One was for Oncternal Therapeutics’ (Nasdaq: ONCT), TK216, an investigational potentially first-in-class targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, for treatment of Ewing sarcoma, with the news pushing the biotech firm’s share up 9%. Ewing sarcoma is the second most common bone tumor among children and adolescents.

Subject to FDA approval of TK216 for the treatment of Ewing sarcoma, Oncternal may be eligible to receive a priority review voucher if the marketing application submitted for the product satisfies certain additional conditions, including approval no later than September 30, 2022 (unless this statutory sunset provision is modified by Congress). If issued, this voucher may be redeemed to receive priority review for a subsequent marketing application or may be sold or transferred to another sponsor.