Clinical Medicine Insights: Therapeutics

In November 2011, the United States Food and Drug Administration (FDA) approved the use of a novel Janus Kinase (JAK) 1/JAK2 inhibitor, INCB 018424 (ruxolitinib), for use in both intermediate and high risk myelofibrosis. Approvals of this agent in both Canada and Europe have followed most recently. The European Medicines Agency (EMA) concluded that ruxolitinib was indicated for disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera (PV) myelofibrosis, and post-essential thrombocythaemia (ET) myelofibrosis. In this review we will consider the rationale for targeting of the JAK-pathway, discuss the pharmacological profile of ruxolitinib and review the currently available clinical trial data. We will also postulate on the current and potential future roles of ruxolitinib within the MPN field.