Novartis reports positive results from Jakavi Phase III trial in patients with polycythemia vera

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 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.

In the trial, a total of 222 patients with PV resistant to or intolerant of hydroxyurea were randomized 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.

At week 32 of the trial, 77% of patients randomized to ruxolitinib achieved one or both components of the composite endpoint of hematocrit control without use of phlebotomy or spleen size reduction in comparison with 20% of patients randomized 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 adopted a positive opinion for ruxolitinib to treat adult patients with PV who are resistant to or intolerant of hydroxyurea.

Currently, Ruxolitinib is approved in more than 70 countries for patients with myelofibrosis, a debilitating and life-threatening blood cancer.

Image: Novartis headquarters in Basel Switzerland. Photo: courtesy of Andrew.