FDA Approves BESREMi, Offering New Essential Thrombocythemia Options
The U.S. Food and Drug Administration (FDA) has approved BESREMi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia (ET), a rare blood cancer that causes the bone marrow to produce too many platelets. The approval, announced by PharmaEssentia USA Corporation, makes BESREMi the first new FDA-approved treatment for essential thrombocythemia in nearly 30 years.
What is Essential Thrombocythemia?
Essential thrombocythemia is a rare blood cancer and a type of myeloproliferative neoplasm, or MPN. In people with ET, the bone marrow produces too many platelets, which can increase the risk of blood clots, abnormal bleeding, and an enlarged spleen. ET is often associated with genetic mutations, including mutations in the JAK2 gene.
ET is a chronic condition that requires long-term monitoring and treatment to help manage the disease and reduce the risk of complications such as stroke, heart attack, and pulmonary embolism.
What Does the FDA Approval of BESREMi Mean for People With ET?
The approval gives adults with essential thrombocythemia another treatment option, including people who are newly diagnosed and have not previously received cytoreductive treatment. BESREMi is a long-acting interferon-based therapy designed to provide sustained disease management, targeting disease-driving cells in the bone marrow while reducing raised platelet counts and disease burden.
“For nearly 30 years, people living with ET and the physicians treating them have had limited treatment innovation,” said Ruben Mesa, M.D., principal investigator of the SURPASS-ET trial. “The approval of BESREMi 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.”
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BESREMi was previously approved and marketed for adults with polycythemia vera (PV), another type of myeloproliferative neoplasm.
How Effective Was BESREMi in Clinical Trials?
The FDA approval was supported by results from the global phase 3 SURPASS-ET clinical trial. BESREMi demonstrated durable modified European Leukemia Net (ELN) responses and durable hematologic control in adults with essential thrombocythemia. The treatment achieved superior durable response rates compared with anagrelide and reduced thromboembolic events over 12 months of treatment.
Research also includes findings from the EXCEED-ET study, which evaluated ropeginterferon alfa-2b-njft in people with ET across different driver mutations.
BESREMi carries a boxed warning due to potential serious side effects, including neuropsychiatric, autoimmune, ischemic, and infectious disorders. It should not be used by people with a history of severe depression, certain liver problems, active serious or untreated autoimmune disease, or certain transplant histories. Doctors may monitor patients for a range of potential complications.
