The European Commission has approved Jakavi (ruxolitinib) from Novartis for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea.
Jakavi is the first targeted treatment approved by the European Commission for these patients.
PV is a rare and incurable blood cancer associated with an overproduction of blood cells that can cause serious cardiovascular complications, such as blood clots, stroke and heart attack. Approximately 25% of patients with PV develop resistance to or intolerance of hydroxyurea and are considered to have uncontrolled disease.
"The European Commission's approval of Jakavi is encouraging news for patients," said Dr. Claire Harrison, study investigator and Consultant Hematologist, Guy's and St. Thomas' NHS Foundation Trust, London. "Jakavi will fill an unmet need as the first treatment shown to significantly improve hematocrit, as well as symptom control and reduce spleen size in patients with polycythemia vera resistant to or intolerant of hydroxyurea."
The approval is based on data from the pivotal Phase III RESPONSE clinical trial demonstrating that a significantly greater proportion of patients achieved the composite primary endpoint of hematocrit control without use of phlebotomy and spleen size reduction, key measures of disease control, when treated with Jakavi compared to best available therapy (21% compared to 1%, respectively; p<0.0001). In the study, a 50% or more improvement in PV-related symptoms was seen in 49% of Jakavi-treated patients compared to 5% of patients treated with best available therapy.
"The approval of Jakavi in polycythemia vera underscores what's possible in today's era of precision oncology research," said Bruno Strigini, President, Novartis Oncology. "Jakavi specifically targets the JAK-STAT pathway, which regulates blood cell production and is known to play a key role in the underlying mechanism of this disease, bringing patients and physicians a new way to treat polycythemia vera."
The European Commission approval applies to all 28 EU member states, plus Iceland, Norway and Liechtenstein. Additional regulatory applications for ruxolitinib in PV are currently ongoing in countries worldwide, and further regulatory filings are under review by health authorities. Ruxolitinib, which is marketed in the US by Incyte Corporation as Jakafi®, received approval in December 2014 from the US Food and Drug Administration (FDA) for the treatment of patients with PV who have had an inadequate response to or are intolerant of hydroxyurea.