The results from a Phase III clinical trial indicate that Jakafi® (ruxolitinib) controlled hematocrit levels and reduced spleen size by 35 percent or more among patients with polycythemia vera.
Myeloproliferative neoplasms (MPN) are a related group of blood cancers. In these disorders, the bone marrow cells that produce blood cells develop and function abnormally. The three main types of MPN are polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). The three conditions are collectively known as myelofibrosis diseases.
One out of four patients with polycythemia vera remain uncontrolled despite existing standard therapies—and these patients must cope with profound symptoms and are at greater risk of cardiovascular complications.
Jakafi—approved in 2011—is currently the only drug that has been approved specifically for myelofibrosis diseases. It is a targeted therapy known as a JAK inhibitor. Jakafi can help to relieve the signs and symptoms of myelofibrosis, such as enlargement of the spleen, night sweats, itching, and bone or muscle pain.
The RESPONSE trial, was a randomized, open-label, phase III study conducted at 109 sites worldwide. It included 222 patients with polycythemia vera resistant to or intolerant of hydroxyurea. Hydroxyurea is in a class of drugs known as antimetabolites and is used to treat cancer and sickle cell anemia by stopping or slowing the growth of blood cells.
Patients were randomly assigned to receive Jakafi (10 mg twice-daily) or best available therapy. The dose was adjusted as needed throughout the trial.
The primary endpoint of the trial is the proportion of patients whose hematocrit level is controlled in absence of phlebotomy and whose spleen volume is reduced by 35 percent or more from baseline as assessed by imaging at 32 weeks. Key secondary endpoints include safety, durable response and complete hematological remission.
Researchers announced that the study met its primary endpoint. What’s more, the safety profile of Jakafi was generally consistent with previous studies based on initial review of the data.
Data from the pivotal phase III trial is expected to be presented at an upcoming scientific meeting. A supplemental new drug application is planned for submission to U.S. Food and Drug Administration this year.
Incyte Announces Positive Top-Line Results from Phase III Study of Ruxolitinib in Patients with Polycythemia Vera. Incyte Press Release. Available at: http://investor.incyte.com/phoenix.zhtml?c=69764&p=irol-newsArticle&ID=1907098&highlight=
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