On September 1, the U.S. Food and Drug Administration (FDA) approved PharmaEssentia's Besremi (ropeginterferon alfa-2b) for adults with essential thrombocythemia (ET). This marks the first new FDA-approved treatment for ET in nearly 30 years.
Essential thrombocythemia is a type of myeloproliferative neoplasm in which the bone marrow produces too many platelets, increasing the risk of potentially serious blood clots, including strokes, heart attacks, and pulmonary embolisms. Some patients may also experience abnormal bleeding or an enlarged spleen. Besremi is a long-acting interferon therapy previously approved for polycythemia vera (PV), another myeloproliferative neoplasm characterized by overproduction of red blood cells. This expanded approval allows use of Besremi in adults with ET regardless of disease status or genetic subtype, including newly diagnosed patients who have not previously received cytoreductive therapy.
The approval is based on results from the global Phase 3 SURPASS-ET study, which compared Besremi with anagrelide in ET patients who had not responded adequately to hydroxyurea or could not tolerate it. Besremi produced higher rates of durable response according to modified European LeukemiaNet criteria, sustained control of blood counts, and fewer thromboembolic events over 12 months of treatment.
Unlike treatments that focus primarily on lowering platelet counts or managing symptoms, Besremi is designed to act on disease-driving cells in the bone marrow. The therapy uses monopegylation technology, which extends the time the drug remains active in the body. Besremi is expected to be available immediately in the United States and was also recently approved for ET in Japan and Taiwan. Like other interferon alfa therapies, Besremi carries a boxed warning for the risk of serious or potentially life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders.
Source: Global Genes / PharmaEssentia | https://globalgenes.org/raredaily/fda-approves-pharmaessentias-besremi-for-rare-blood-cancer/