The US Food and Drug Administration (FDA) has approved Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia (ET).
ET is a condition characterized by an overproduction of platelets. Abnormal blood clotting is common in these patients and may cause strokes or temporary stroke-like episodes, leg pain, swelling, pulmonary embolism, chest pain and difficulty breathing. A parodoxical problem in ET is abnormal bleeding, such as excessive nosebleeds, bleeding gums, or bleeding in the gastrointestinal tract. Other signs and symptoms include splenomegaly, weakness, headaches, or a sensation in the skin of burning, tingling, or prickling. Some people with ET have episodes of severe pain, redness, and swelling, which commonly occur in the hands and feet.
Ropeginterferon alfa is a long-acting interferon-based therapy designed to target disease-driving cells in the bone marrow while reducing elevated platelet counts and disease burden. It is approved for adults with ET, regardless of their genotype or disease status, including newly diagnosed patients who are naive to cytoreductive therapy. This approval expands the product label, which has been commercially available for the treatment of adults with polycythemia vera (PV).
The approval is based on clinical data from the phase 3 SURPASS ET clinical trial (NCT04285086). In this study, ropeginterferon alfa demonstrated durable modified European Leukemia Net (ELN) responses and durable hematologic control in adults with ET. Superior durable response rates were also achieved, compared with anagrelide, and reduced thromboembolic events over 12 months of treatment.
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To learn more about rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/