Credit: Zak Hubbard
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending the approval of ruxolitinib (Jakavi) to
treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
The European Commission generally follows the CHMP’s advice and delivers its final decision within 3 months of the CHMP recommendation.
If approved, ruxolitinib would be the first targeted treatment option for PV patients in Europe.
The European Commission’s decision will be applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About ruxolitinib
Ruxolitinib is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. It was approved by the European Commission in August 2012 to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF. The drug is approved to treat MF in more than 70 countries.
Novartis licensed ruxolitinib from Incyte Corporation (which markets the drug as Jakafi in the US) for development and commercialization outside the US. Ruxolitinib is approved in the US to treat patients with MF and those with PV.
Ruxolitinib in PV: The RESPONSE trial
The CHMP’s recommendation to approve ruxolitinib for PV was based on results from the phase 3 RESPONSE trial, which was funded by Incyte.
The trial included 222 patients who had PV for at least 24 weeks. All patients had an inadequate response to or could not tolerate hydroxyurea, had undergone a phlebotomy, and had an enlarged spleen.
They were randomized to receive ruxolitinib starting at 10 mg twice daily or best available therapy (BAT) as determined by the investigator. The ruxolitinib dose was adjusted as needed.
The study was designed to measure the reduced need for phlebotomy beginning at week 8 and continuing through week 32, in addition to at least a 35% reduction in spleen volume at week 32.
Twenty-one percent of ruxolitinib-treated patients met this endpoint, compared to 1% of patients who received BAT.
Ruxolitinib was generally well-tolerated, but 3.6% of patients discontinued treatment due to adverse events, compared to 1.8% of patients on BAT.
The most common hematologic adverse events associated with ruxolitinib were anemia and thrombocytopenia. The most common non-hematologic events were headache, diarrhea, fatigue, dizziness, constipation, and shingles.
