Today, the U.S. Food and Drug Administration (FDA) approved ropeginterferon alfa-2b-njft (Besremi) for the treatment of adults with essential thrombocythemia, making it the first new FDA-approved treatment for essential thrombocythemia in nearly 3 decades. Ropeginterferon alfa-2b-njft has been approved for adults with essential thrombocythemia regardless of their genotype or disease status, including newly diagnosed patients who are naive to cytoreductive therapy. The essential thrombocythemia approval expands the ropeginterferon alfa-2b-njft product label, which has been commercially available for the treatment of adults with polycythemia vera.
The FDA approval of ropeginterferon alfa-2b-njft is based on clinical data demonstrating durable modified European Leukemia Net responses and durable hematologic control in adults with essential thrombocythemia. In the global phase III SURPASS ET clinical trial (ClinicalTrials.gov identifier NCT04285086), ropeginterferon alfa-2b-njft achieved superior durable response rates compared with anagrelide and reduced thromboembolic events over 12 months of treatment.
This approval marks a major milestone for people living with essential thrombocythemia. Patients with essential thrombocythemia often require years of ongoing disease management and remain at risk for serious complications, such as heart attacks, strokes, and pulmonary embolism.
Ropeginterferon alfa-2b-njft is a long-acting interferon-based therapy designed to provide sustained disease management for essential thrombocythemia. It offers a differentiated treatment option compared with current therapies that primarily focus on controlling platelet counts and symptoms, with limited impact on the underlying disease biology. Using novel monopegylation technology and an extended half-life, ropeginterferon alfa-2b-njft is designed to target disease-driving cells in the bone marrow while reducing elevated platelet counts and disease burden.
“For nearly 30 years, people living with [essential thrombocythemia] and the physicians treating them have had limited treatment innovation,” said Ruben Mesa, MD, Principal Investigator of the SURPASS-ET trial and President of Advocate Health’s Cancer National Service Line, which includes Atrium Health Levine Cancer Institute and the Comprehensive Cancer Center at Atrium Health Wake Forest Baptist in Winston-Salem, North Carolina. “In my experience, patients need treatment options that not only control blood counts but also address the underlying disease. The approval of ropeginterferon alfa-2b-njft provides an important new treatment option that is supported by strong clinical evidence and that also works at the source of the disease rather than solely managing symptoms.”
