On March 7, 2014 Novartis reported that a pivotal Phase III trial of Jakavi (ruxolitinib) compared to best available therapy has met its primary endpoint of maintaining hematocrit control without the need for phlebotomy and reducing spleen size in patients with polycythemia vera resistant to or intolerant of hydroxyurea (Press release Novartis, MAR 6, 2014, View Source [SID:1234500248]). The safety profile of ruxolitinib was generally consistent with previous studies based on initial review of the data.
Data from the study (RESPONSE) will be presented at an upcoming medical congress and submitted to worldwide regulatory authorities this year.
Novartis plans to submit these data to worldwide regulatory agencies this year, as they seek to bring ruxolitinib to patients with polycythemia vera who are no longer responding to or are intolerant of prior therapy.
RESPONSE is a global, randomized, open-label study conducted at 109 sites. The trial randomized 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea. Patients were randomized 1:1 to receive either ruxolitinib (10 mg twice-daily) or best available therapy, which was defined as investigator selected monotherapy or observation only. The dose was adjusted as needed throughout the study.
The primary endpoint of the study is the proportion of patients whose hematocrit is controlled without phlebotomy and whose spleen volume is reduced by 35% or more from baseline as assessed by imaging at 32 weeks. In addition to safety, key secondary endpoints include durable response and complete hematological remission.
Ruxolitinibis currently approved in more than 55 countries for patients with myelofibrosis, a debilitating and life-threatening blood cancer.