The MPN Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

New drug application (NDA) for accelerated FDA approval to be submitted for pacritinib in treating myelofibrosis with severe thrombocytopenia

Sep 30, 2020

The U.S Food and Drug Administration (FDA) has agreed to a new drug application (NDA) submission which may lead to an accelerated approval of pacritinib, a Janus kinase 2 (JAK2) and interleukin-1-receptor-associated kinase 1 (IRAK1) inhibitor, for the treatment of patients with myelofibrosis (MF) with severe thrombocytopenia (platelet < 50 × 10 9/L).

The application will be based on the phase III PERSIST-1and PERSIST-2trials where pacritinib has shown clinical superiority to best available therapy when treating JAK inhibitor-naïve patients with primary MF and thrombocytopenia, and the phase II PAC203, comparing the efficacy and safety of different pacritinib dosing schedules in patients with MF, who no longer benefitted or were intolerant to JAK inhibitors. The submission is expected to be completed in early 2021. In addition, pacritinib is currently under investigation in the phase III PACIFICAtrial comparing the efficacy of 200 mg pacritinib with the physician’s choice in patients with primary or secondary MF.

For more information on the management of thrombocytopenia in patients with MF read here.

  1. BioSpace. CTI BioPharma to submit a new drug application (NDA) for the accelerated approval of pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia. https://www.biospace.com/article/releases/cti-biopharma-to-submit-a-new-drug-application-nda-for-the-accelerated-approval-of-pacritinib-for-the-treatment-of-myelofibrosis-patients-with-severe-thrombocytopenia/. Published Sep 29, 2020. Accessed Sep 30, 2020.