Novartis has reported positive results from its pivotal Phase III clinical trial of Jakavi (ruxolitinib), an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases, for the treatment of patients with polycythemia vera (PV).
The results showed that Jakavi improved hematocrit control without the need for phlebotomy and reduced spleen size in PV patients who had an inadequate response to or unacceptable side effects from hydroxyurea as defined according to the modified European LeukemiaNet (ELN) criteria.
Phlebotomy is a procedure to remove blood from the body in order to reduce the concentration of red blood cells.
Hematocrit control and spleen size reduction are key measures of a PV patient’s response to therapy.
During the trial, a total of 222 patients with PV resistant to or intolerant of hydroxyurea were randomised 1:1 to receive either ruxolitinib (starting dose of 10mg twice daily) or standard therapy, which was defined as investigator-selected monotherapy or observation only.
Azienda Ospedaliera Universitaria Careggi, University of Florence, Italy and lead study author Dr Alessandro Vannucchi said: "A key challenge in treating patients with PV is the development of resistance or intolerance to currently available therapies such as hydroxyurea, which leaves us with very limited alternative treatment options to effectively manage the disease.
"This study indicates that ruxolitinib may represent an important advance for this population of patients with PV, a disease that can lead to serious complications and difficult daily symptoms."
At week 32 of the trial, 77% of patients treated with ruxolitinib achieved one or both components of the composite endpoint of hematocrit control without use of phlebotomy or spleen size reduction, compared to 20% of patients who were randomised to standard therapy.
Novartis Oncology Development and Medical Affairs global head Alessandro Riva said: "A high unmet need exists for PV patients with uncontrolled disease on current treatments, and if approved in the EU, ruxolitinib is expected to be the first-ever targeted therapy for patients with PV.
"We are working diligently with regulatory authorities to bring ruxolitinib to patients with PV who may benefit from this treatment."
Based on the Phase III data, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recently adopted a positive opinion for ruxolitinib to treat adult patients with PV who are resistant to or intolerant of hydroxyurea.
Ruxolitinib is currently approved in more than 70 countries for patients with myelofibrosis, including the EU, Canada, Japan and some countries in Asia, Latin and South America.