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

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 × 10⁹/L).

The application will be based on the phase III PERSIST-1 and PERSIST-2 trials 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 PACIFICA trial 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.