As reported on Pharmaceutical Technology, the US Food and Drug Administration (FDA) has granted approval to PharmaEssentia’s Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythaemia (ET), providing patients with the first newly approved therapy for the disease in almost three decades.
The decision was supported by findings from the global Phase III SURPASS ET study, which demonstrated that Besremi delivered sustained clinical responses and long-term control of key blood-count parameters in adults living with ET.
Under the expanded label, Besremi can be used across the broad ET patient population, regardless of genetic mutation status or stage of disease. The treatment is also available to both newly diagnosed patients and those who have not previously received cytoreductive therapy.
The approval represents an important milestone for PharmaEssentia, extending the drug’s US indications beyond polycythaemia vera (PV), for which Besremi was already authorized.
Essential thrombocythaemia is a rare myeloproliferative neoplasm characterized by excessive production of platelets in the bone marrow. The condition can increase the likelihood of serious thrombotic complications, including stroke, myocardial infarction, and pulmonary embolism.
Besremi is a long-acting interferon therapy designed to provide durable disease control. The treatment targets abnormal blood-forming cells in the bone marrow while helping to reduce elevated platelet levels associated with ET.
Results from the SURPASS ET trial showed that patients receiving Besremi achieved higher rates of durable response compared with those treated with anagrelide. The study also reported fewer thromboembolic events among patients receiving Besremi during the 12-month treatment period.
Commenting on the approval, PharmaEssentia founder and chief executive officer Ko-Chung Lin said the FDA’s decision marks a meaningful advance for individuals living with ET and reflects the company’s ongoing commitment to developing therapies for patients with myeloproliferative neoplasms. He added that PharmaEssentia has worked with regulators, healthcare professionals, patient advocacy groups, and payers to support access to the treatment.
The US authorization follows recent regulatory approvals for Besremi in Japan and Taiwan for the same indication. PharmaEssentia, which is headquartered in Taiwan and operates across Asia and the United States, maintains worldwide intellectual property rights for the drug. Besremi also holds orphan drug designation in the US for both polycythaemia vera and essential thrombocythaemia.
