Loading Curie Briefs...
Getting the latest healthcare news for you
Getting the latest healthcare news for you
A long-awaited breakthrough for a rare blood disorder. The FDA has approved ropeginterferon alfa-2b-njft (Besremi) for adults with essential thrombocythemia (ET) — the first new treatment for the condition in almost three decades. In the pivotal SURPASS ET trial, Besremi outperformed anagrelide with a 37.4% vs. 3.6% durable response rate, and it's indicated regardless of genotype or prior treatment status.
A long-awaited breakthrough for a rare blood disorder
The FDA has approved ropeginterferon alfa-2b-njft (Besremi, PharmaEssentia) for adults with essential thrombocythemia (ET), marking the first new treatment approval for this rare blood cancer in nearly 30 years. ET causes the bone marrow to overproduce platelets, raising the risk of dangerous blood clots and bleeding. The approval covers all adults regardless of genotype or disease status — including newly diagnosed patients who haven't yet received cytoreductive therapy.
The drug, a long-acting interferon already approved for polycythemia vera, is designed to target the disease at its source rather than just managing symptoms. It's given as a subcutaneous injection, starting at 250 µg and titrating up to a maintenance dose of 500 µg every two weeks.
By the Numbers
Why it matters: With no new ET therapies approved since the late 1990s, Besremi fills a critical gap for patients who have had inadequate responses or intolerance to hydroxyurea. Its disease-modifying mechanism — rather than symptom control alone — could reshape how clinicians approach ET management.
