Burlington, Mass. — PharmaEssentia USA Corporation announced that the U.S. Food and Drug Administration has approved BESREMi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia, or ET, marking the first new FDA-approved treatment for the rare blood cancer in nearly three decades.
The approval covers adults with ET regardless of genotype or disease status, including newly diagnosed patients who have not previously received cytoreductive therapy. It expands the label for BESREMi, which is already approved in the United States for adults with polycythemia vera.
Essential thrombocythemia is a chronic blood cancer characterized by the overproduction of platelets. Patients may require years of disease management and face an increased risk of complications including heart attacks, strokes and pulmonary embolism.
BESREMi is a long-acting interferon-based therapy designed to provide sustained disease control. PharmaEssentia said the treatment is intended to target disease-driving cells in the bone marrow while reducing elevated platelet counts and overall 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 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 my experience, patients need treatment options that not only control blood counts but also address the underlying disease. 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.”
The FDA approval was based on results from the global Phase 3 SURPASS ET clinical trial, which evaluated BESREMi against anagrelide. According to PharmaEssentia, BESREMi demonstrated superior durable response rates and reduced thromboembolic events over 12 months of treatment.
The study also showed durable modified European Leukemia Net responses and sustained hematologic control in adults with ET.
“Today’s FDA approval of BESREMi for ET is a significant advancement for patients and reflects PharmaEssentia’s longstanding commitment to advancing innovative therapies for people living with myeloproliferative neoplasms,” said Ko-Chung Lin, Ph.D., founder and CEO of PharmaEssentia. “We have been working closely with the FDA, healthcare providers, advocacy organizations and payers to bring this important treatment option to the ET community.”
The approval follows recent regulatory clearances for BESREMi in Japan and Taiwan for ET. PharmaEssentia said the treatment is expected to be available in the United States immediately.
