Pacritinib: First Approval

Pacritinib (VONJO (TM)) is an orally administered, small molecule kinase inhibitor being developed by CTI BioPharma for the treatment of myelofibrosis and graft-versus-host disease. Pacritinib received its first approval in February 2022 in the USA 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 x 10(9)/L. The accelerated approval was based on results from the randomized, active-controlled, phase III PERSIST-2 trial, in which spleen volume reduction was demonstrated in pacritinib recipients. This article summarizes the milestones in the development of pacritinib leading to this first approval for myelofibrosis.

Automated summary

Pacritinib is an orally administered kinase inhibitor developed for the treatment of myelofibrosis and graft-versus-host disease. It has received accelerated approval in the USA for the treatment of specific types of myelofibrosis based on clinical trial results.