Novartis gets EU approval for Jakavi® in polycythemia vera

Novartis has announced that the European Commission has approved Jakavi® (ruxolitinib) 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. It is the only JAK 1/2 inhibitor available to treat PV and is also currently approved in more than 80 countries to treat myelofibrosis.

PV is a rare and incurable blood cancer associated with an overproduction of blood cells that, if left untreated, can lead to 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. This is typically defined as hematocrit levels greater than 45%, elevated white blood cell count and/or platelet count, and may be accompanied by debilitating symptoms and/or an enlarged spleen.

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.

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.

Dr. Claire Harrison, study investigator and Consultant Haematologist, Guy’s and St. Thomas’ NHS Foundation Trust, London, said: “The European Commission’s approval of Jakavi is encouraging news for patients. 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.”