Novartis has presented clinical data showing that its Jakavi was superior to best available therapy in patients with the rare haematological cancer polycythemia vera (PV), which affects around 100,000 people globally a year.
According to findings of the Phase III RESPONSE-2 study, Jakavi (ruxolitinib) helped patients with certain patients PV achieve superior hematocrit control - the ratio of the volume of red blood cells to the total volume of blood - compared to best available therapy (BAT) at 28 weeks (62.2 percent vs 18.7 percent, respectively).
Polycythemia vera is a rare and incurable blood cancer associated with an overproduction of blood cells that can cause serious cardiovascular complications, including blood clots, stroke and heart attack. As the disease progresses, the spleen can become enlarged as it works to clear a greater number of blood cells than normal.
Patients involved in this study did not have an enlarged spleen and were resistant to or intolerant of hydroxyurea, and so were considered to have a less advanced stage of PV. The data "support the use of Jakavi as a second-line treatment option to help this patient population gain better control of their disease," noted lead study investigator, Francesco Passamonti, MD, the University of Insubria, Varese, Italy.
As well as hitting its primary endpoint, the trial also showed that nearly five times more patients with PV achieved complete haematologic remission with Jakavi compared to BAT at 28 weeks (23 percent vs 5.3 percent, respectively).
More patients taking Novartis' drug experienced complete resolution of their symptoms related to PV compared to BAT (50.0 percent vs 7.7 percent, respectively) and the drug giant said that Jakavi was well tolerated in the patient group.
Meanwhile, in more positive news on the drug, Phase III data from the COMFORT-I study, also presented at the 21st Congress of the European Haematology Association (EHA) in Copenhagen, Denmark, indicate an overall survival advantage in patients with intermediate-2 or high-risk myelofibrosis (MF) taking to Jakavi compared to those given a placebo.
The five-year survival showed a 31 precent reduction in the risk of death in the Jakavi arm despite more than 70 percent of patients randomised to the placebo arm crossing over to receive treatment with the drug, Novartis said, noting that the findings support its "durable efficacy and long-term safety profile" in MF.Jakavi, an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases licensed rom Incyte, is approved in Europe for adults with PV who are resistant to/intolerant of hydroxyurea, and for the treatment of disease-related splenomegaly or symptoms in adults with various types of MF.