The U.S. Food and Drug Administration (FDA) has approved ropeginterferon alfa-2b-njft (Besremi) for the treatment of polycythemia vera.
Polycythemia vera is a rare blood disorder characterized by an increased concentration of blood cells. In most cases, polycythemia vera is acquired rather than congenital, and it predominantly affects older adults. It is associated with mutations in the JAK2 and TET2 genes. While patients can live for decades with this disorder, there is an increased risk of thrombotic events. Symptoms of this disorder are varied and may include fevers, chills, itching, and fatigue. The most common treatment for polycythemia vera includes frequent phlebotomies.
Ropeginterferon alfa-2b-njft is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history, and the first interferon therapy specifically approved for polycythemia vera. Ropeginterferon alfa-2b-njft is believed to act by reducing blood cell production in the bone marrow.
The approval of ropeginterferon alfa-2b-njft was based on positive results of a multicenter, single arm trial, which evaluated the drug’s safety and effectiveness. Effectiveness was assessed by the number of patients who achieve complete hematological response. Overall, 61% of patients had a complete hematological response.
To learn more about polycythemia vera and other rare blood disorders, visit checkrare.com/diseases/hematology