Novartis reports positive Phase III study for Jakavi in polycythemia vera patients

Polycythemia vera a chronic, incurable blood cancer associated with an overproduction of blood cells.

Novartis announced that the Phase III study of Jakavi has met its primary endpoint of maintaining hematocrit control (red blood cell volume) without the need for phlebotomy and reducing spleen size in patients with polycythemia vera resistant to or intolerant of hydroxyurea.

The Phase III study (RESPONSE) demonstrated generally consistent safety profile of ruxolitinib with previous studies based on initial review of the data

The pivotal, randomized trial was conducted at 109 sites. A total of 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea were randomized 1:1 to receive either ruxolitinib (10mg twice-daily) or best available therapy that was defined as investigator selected monotherapy or observation only. Dose was adjusted as needed throughout the study.

Primary endpoint of the open-label 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. The secondary endpoints include safety, durable response and complete hematological remission.

The European Commission has approved Jakavi in August 2012 for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

Jakavi is currently approved in over 55 countries including the EU, Canada and some countries in Asia, Latin and South America for patients with myelofibrosis, a debilitating and life-threatening blood cancer. Additional worldwide regulatory filings are underway.

Ruxolitinib has been licensed by Novartis from Incyte for development and commercialization outside the US. Jakavi is a registered trademark of Novartis in countries outside the US while Jakafi is a registered trademark of Incyte. Incyte is marketing Jakavi in the US under the name Jakafi for the treatment of patients with intermediate or high-risk myelofibrosis.

The drug has previously received orphan drug status for myelofibrosis from European Commission and the US Food and Drug Administration.

Safety and efficacy profile of Jakavi is yet to be established outside the approved indication.

Novartis Oncology president and Oncology Development and Medical Affairs global head Alessandro Riva noted the company is encouraged by these pivotal Phase III trial results, which show the potential of ruxolitinib to help patients with polycythemia vera.

"We plan to submit these data to worldwide regulatory agencies this year, as we seek to bring ruxolitinib to patients with polycythemia vera who are no longer responding to or are intolerant of prior therapy," Riva added.

Novartis will present the study data at an upcoming medical congress and submitted to worldwide regulatory authorities in 2014.

Image: Novartis AG headquarters in Basel. Photo: Courtesy of Leoboudv