Novartis’ Jakavi for Rare Blood Cancer Receives EU Approval

The European Medicines Agency Tuesday approved Novartis’ Jakavi for patients with polycythemia vera — a rare blood cancer — who cannot take hydroxyurea.

Jakavi (ruxolitinib) is the first targeted treatment for PV in the EU, Novartis says.

In a pivotal trial of 222 patients across 90 sites, 21 percent in the Jakavi arm were able to control the volume of red blood cells in their blood without transfusions or reducing the size of the spleen. By contrast, only 1 percent of patients randomized to the best available therapy arm achieved this aim.

In addition, nearly half of those receiving Jakavi saw better than 50 percent improvement in symptoms such as headache, dizziness, double or blurred vision, pressure from an enlarged spleen or bleeding heavily from small cuts, compared with 5 percent of controls.

Jakavi is already available in Europe for adults with primary myelofibrosis, post-PV myelofibrosis and post-essential thrombocythemia myelofibrosis. Novartis licensed the drug to Incyte in the U.S., where it won FDA approval as Jakafi in December. — Lena Freund