Novartis Posts Positive Phase 3 Data For Polycythemia Vera Drug

Novartis announced positive results from the first pivotal Phase III trial evaluating Jakavi (ruxolitinib) as treatment for polycythemia vera (PV).

Polycythemia vera is a chronic blood cancer linked with uncontrollable production of blood cells in the bone marrow. Currently, there are few PV treatments available. The disease affects about one to three people per 100,000 around the world. PV is characterized by an increase in the volume percentage of red blood cells, or elevated hematocrit. This typically leads to blood thickening and increased risk of blood clots, which can result in serious cardiovascular complications.

Jakavi (ruxolitinib) is an oral JAK 1 and JAK 2 tyrone kinases inhibitor. The drug was approved by the FDA in 2011 as a treatment for myelofibrosis. The European Commission also approved the drug in August 2012 as treatment for disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Novartis licensed the drug from Incyte Corporation for development and marketing outside the U.S.

The global, randomized, open-label RESPONSE study showed that Jakavi significantly improved hematocrit control without need for the standard phlebotomy. The drugs also reduced spleen size in patients with PV who are intolerant or have developed resistance to hydroxyurea. Seventy-seven percent of patients who received Jakavi achieved the primary endpoint of the trial compared to 20 percent on best available therapy. Almost half of ruxolitinib-treated patients had a 50 percent or more reduction in debilitating PV symptoms compared to 5 percent of the second group taking another therapy.

“In the RESPONSE trial, patients treated with ruxolitinib showed better disease control, including controlled hematocrit levels without the need for phlebotomy, reduced spleen size and improved symptom management compared to current therapies,” said Srdan Verstovsek, of MD Anderson Cancer Center in Houston, Texas and lead author of the study.

The company said that regulatory filings around the world are underway based on the trial data. If approved, ruxolitinib will be the first JAK 1 and JAK 2 inhibitor for patients with polycythemia vera.