Incyte Corp (INCY) said Thursday the U.S. Food and Drug Administration has approved Jakafi (ruxolitinib) for the treatment of patients with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea. Jakafi, an oral medication, is the first and only product approved by the FDA for PV, a rare and progressive blood cancer.
PV is a myeloproliferative neoplasm (MPN) and is typically characterized by elevated hematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots, as well as an elevated white blood cell and platelet count. PV may occur at any age but often presents later in life, with a median age at diagnosis of 60 years. About 100,000 patients in the U.S. are living with PV.
Current standard treatment for PV is phlebotomy (the removal of blood from the body) plus aspirin. When phlebotomy can no longer control PV, chemotherapy such as hydroxyurea, or interferon, is utilized. Approximately one in four patients with PV are considered uncontrolled because they have an inadequate response to or are intolerant of hydroxyurea, the most commonly used chemotherapeutic agent for the treatment of PV.
Jakafi is also the first and only FDA-approved product for the treatment of intermediate or high-risk myelofibrosis, a closely related blood cancer. Jakafi is a JAK1 and JAK2 inhibitor that targets overactive JAK pathway signaling, which plays a critical role in the development of both myelofibrosis and polycythemia vera10.
The approval of Jakafi for the treatment of patients with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea was based on data from the pivotal Phase III RESPONSE trial, which was conducted under a Special Protocol Assessment from the FDA.
In this trial, patients treated with Jakafi demonstrated superior hematocrit control and reductions in spleen volume compared to best available therapy. In addition, a greater proportion of patients on the ruxolitinib treatment arm achieved complete hematologic remission - which was defined as achieving hematocrit control, and lowering platelet and white blood cell counts.
In the RESPONSE trial, the most common hematologic adverse reactions were thrombocytopenia and anemia. The most common non-hematologic adverse events were headache, abdominal pain, diarrhea, dizziness, fatigue, pruritus, dyspnea and muscle spasms.
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.
Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF.
Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States.
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