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The European Commission (EC) has approved ruxolitinib (Jakavi) to treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
This is the first targeted treatment the EC has approved for these patients.
The approval applies to all 28 member states of the European Union (EU), plus Iceland, Norway, and Liechtenstein.
Ruxolitinib is already approved to treat PV in the US, and additional regulatory applications for ruxolitinib in PV are ongoing worldwide.
The drug is also approved to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF in more than 70 countries, including EU member states and the US.
“The European Commission’s approval of Jakavi [for PV] is encouraging news for patients,” said Claire Harrison, MD, a consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, England.
“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.”
RESPONSE trial
The EC’s approval is based on data from the phase 3 RESPONSE trial. The study 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 splenomegaly.
Patients 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’s primary endpoint was a composite of hematocrit control and spleen reduction. To meet the endpoint, patients had to experience a 35% or greater reduction in spleen volume from baseline, as assessed by imaging at week 32.
And a patient’s hematocrit was considered under control if he was not eligible for phlebotomy from week 8 through 32 (and had no more than one instance of phlebotomy eligibility between randomization and week 8). Patients who were deemed eligible for phlebotomy had hematocrit that was greater than 45% or had increased 3 or more percentage points from the time they entered the study.
Twenty-one percent of ruxolitinib-treated patients met this endpoint (achieving hematocrit control and spleen reduction), compared to 1% of patients who received BAT (P<0.001).
And the researchers said ruxolitinib was well-tolerated. Common adverse events included headache, diarrhea, and fatigue.
Grade 3/4 anemia, grade 3/4 thrombocytopenia, and herpes zoster infections of all grades were more common in the ruxolitinib arm than the BAT arm. But thromboembolic events were more common with BAT than ruxolitinib.
This trial was funded by the Incyte Corporation, which markets ruxolitinib in the US. Novartis licensed ruxolitinib from Incyte for development and commercialization outside the US.
For more details on ruxolitinib, see the full prescribing information, available at www.jakavi.com. 
Image courtesy of AFIP
The European Commission (EC) has approved ruxolitinib (Jakavi) to treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
This is the first targeted treatment the EC has approved for these patients.
The approval applies to all 28 member states of the European Union (EU), plus Iceland, Norway, and Liechtenstein.
Ruxolitinib is already approved to treat PV in the US, and additional regulatory applications for ruxolitinib in PV are ongoing worldwide.
The drug is also approved to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF in more than 70 countries, including EU member states and the US.
“The European Commission’s approval of Jakavi [for PV] is encouraging news for patients,” said Claire Harrison, MD, a consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, England.
“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.”
RESPONSE trial
The EC’s approval is based on data from the phase 3 RESPONSE trial. The study 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 splenomegaly.
Patients 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’s primary endpoint was a composite of hematocrit control and spleen reduction. To meet the endpoint, patients had to experience a 35% or greater reduction in spleen volume from baseline, as assessed by imaging at week 32.
And a patient’s hematocrit was considered under control if he was not eligible for phlebotomy from week 8 through 32 (and had no more than one instance of phlebotomy eligibility between randomization and week 8). Patients who were deemed eligible for phlebotomy had hematocrit that was greater than 45% or had increased 3 or more percentage points from the time they entered the study.
Twenty-one percent of ruxolitinib-treated patients met this endpoint (achieving hematocrit control and spleen reduction), compared to 1% of patients who received BAT (P<0.001).
And the researchers said ruxolitinib was well-tolerated. Common adverse events included headache, diarrhea, and fatigue.
Grade 3/4 anemia, grade 3/4 thrombocytopenia, and herpes zoster infections of all grades were more common in the ruxolitinib arm than the BAT arm. But thromboembolic events were more common with BAT than ruxolitinib.
This trial was funded by the Incyte Corporation, which markets ruxolitinib in the US. Novartis licensed ruxolitinib from Incyte for development and commercialization outside the US.
For more details on ruxolitinib, see the full prescribing information, available at www.jakavi.com.
Image courtesy of AFIP
The European Commission (EC) has approved ruxolitinib (Jakavi) to treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
This is the first targeted treatment the EC has approved for these patients.
The approval applies to all 28 member states of the European Union (EU), plus Iceland, Norway, and Liechtenstein.
Ruxolitinib is already approved to treat PV in the US, and additional regulatory applications for ruxolitinib in PV are ongoing worldwide.
The drug is also approved to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF in more than 70 countries, including EU member states and the US.
“The European Commission’s approval of Jakavi [for PV] is encouraging news for patients,” said Claire Harrison, MD, a consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, England.
“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.”
RESPONSE trial
The EC’s approval is based on data from the phase 3 RESPONSE trial. The study 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 splenomegaly.
Patients 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’s primary endpoint was a composite of hematocrit control and spleen reduction. To meet the endpoint, patients had to experience a 35% or greater reduction in spleen volume from baseline, as assessed by imaging at week 32.
And a patient’s hematocrit was considered under control if he was not eligible for phlebotomy from week 8 through 32 (and had no more than one instance of phlebotomy eligibility between randomization and week 8). Patients who were deemed eligible for phlebotomy had hematocrit that was greater than 45% or had increased 3 or more percentage points from the time they entered the study.
Twenty-one percent of ruxolitinib-treated patients met this endpoint (achieving hematocrit control and spleen reduction), compared to 1% of patients who received BAT (P<0.001).
And the researchers said ruxolitinib was well-tolerated. Common adverse events included headache, diarrhea, and fatigue.
Grade 3/4 anemia, grade 3/4 thrombocytopenia, and herpes zoster infections of all grades were more common in the ruxolitinib arm than the BAT arm. But thromboembolic events were more common with BAT than ruxolitinib.
This trial was funded by the Incyte Corporation, which markets ruxolitinib in the US. Novartis licensed ruxolitinib from Incyte for development and commercialization outside the US.
For more details on ruxolitinib, see the full prescribing information, available at www.jakavi.com.