CTI BioPharma Granted FDA Accelerated Approval of Vonjo (pacritinib) for the Treatment of Myelofibrosis and Thrombocytopenia
CTI BioPharma announced the U.S FDA has granted accelerated approval to Vonjo (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
The FDA previously granted priority review, fast track designation and orphan drug designation. Vonjo is a novel oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1.
FDA accelerated approval was based on efficacy results from the pivotal Phase 3 PERSIST-2 study of Vonjo in patients with myelofibrosis (platelet counts less than or equal to 100 × 109/L). Patients were randomized 1:1:1 to receive Vonjo 200 mg twice daily (BID), Vonjo 400 mg once daily (QD) or best available therapy (BAT).
Prior JAK2 inhibitor therapy was permitted. In this study, in the cohort of patients with baseline platelet counts below 50 × 109/L who were treated with Vonjo 200 mg BD, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib.
March 4, 2022
