The U.S. Food and Drug Administration (FDA) has approved PharmaEssentia’s BESREMi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia (ET), making it the first new FDA-approved treatment for the rare blood cancer in nearly three decades.
The approval expands the use of BESREMi, which is already available in the United States for adults with polycythemia vera (PV). The new approval covers adults with ET regardless of their genotype or disease status, including newly diagnosed patients who have not previously received cytoreductive therapy.
Essential thrombocythemia is a chronic blood cancer in which the bone marrow produces too many platelets. The condition can increase the risk of serious complications, including blood clots, heart attacks, strokes and pulmonary embolism.
BESREMi is a long-acting interferon-based treatment designed to provide sustained control of the disease. Unlike therapies that mainly focus on lowering platelet counts and managing symptoms, BESREMi is designed to target disease-driving cells in the bone marrow and reduce overall disease burden.
The FDA approval was supported by results from the global Phase 3 SURPASS-ET trial. In the study, BESREMi demonstrated durable modified European LeukemiaNet (ELN) responses and sustained control of blood counts in adults with essential thrombocythemia.
The trial also showed that patients treated with BESREMi had superior durable response rates compared with anagrelide. The treatment was also associated with a reduction in thromboembolic events over 12 months of treatment.
Ruben Mesa, M.D., principal investigator of the SURPASS-ET trial, said the new approval provides an important option for patients who need treatments that go beyond controlling blood counts and symptoms.
PharmaEssentia founder and CEO Ko-Chung Lin, Ph.D., said the approval represents an important milestone in the company’s efforts to develop new treatments for people living with myeloproliferative neoplasms.
BESREMi uses the company’s monopegylation technology, which gives the treatment an extended half-life and allows it to provide longer-lasting activity. The therapy is intended to target cells involved in the development of essential thrombocythemia while helping control elevated platelet levels.
The FDA decision follows recent regulatory approvals for BESREMi in Japan and Taiwan for essential thrombocythemia, further expanding the treatment’s use across different markets.
PharmaEssentia USA said BESREMi for essential thrombocythemia is expected to be available to patients in the United States immediately following the FDA approval.
The approval provides a new treatment option for adults with essential thrombocythemia and marks a major development in a disease area that has seen limited treatment innovation for many years.