Jakavi is the first targeted treatment approved by the EC for these patients in the European Union (EU) who are resistant to or intolerant of hydroxyurea.
The European approval is based on data from the pivotal Phase III RESPONSE clinical trial, which showed that a significantly greater proportion of patients achieved the composite primary endpoint of hematocrit control without use of phlebotomy and spleen size reduction when treated with Jakavi compared to best available therapy.
The global, randomized, open-label trial carried out at more than 90 trial sites included a total of 222 patients with PV resistant to or intolerant of hydroxyurea and they were randomized 1:1 to receive either Jakavi or best available therapy.
PV is associated with an overproduction of blood cells that can cause serious cardiovascular complications, such as blood clots, stroke and heart attack.
Guy’s and St. Thomas’ NHS Foundation Trust consultant hematologist and study investigator Dr Claire Harrison said: "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."
During the trial, a 50% or more improvement in PV-related symptoms was observed in 49% of patients treated with Jakavi compared to 5% of patients treated with best available therapy.
Novartis Oncology president Bruno Strigini said: "The approval of Jakavi in polycythemia vera underscores what’s possible in today’s era of precision oncology research.
"Jakavi specifically targets the JAK-STAT pathway, which regulates blood cell production and is known to play a key role in the underlying mechanism of this disease, bringing patients and physicians a new way to treat polycythemia vera."
The new European approval applies to all 28 EU member states, plus Iceland, Norway and Liechtenstein.
Currently, additional regulatory applications for ruxolitinib in PV are ongoing in countries worldwide, and further regulatory filings are under review by health authorities.
Image: Novartis headquarters in Basel Switzerland. Photo: courtesy of Andrew.