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Jump to Key Points The FDA has approved ropeginterferon alfa-2b injection (BESREMi, PharmaEssentia) for the treatment of adults with essential thrombocythemia (ET).
Ropeginterferon is the first new FDA-approved treatment for ET in nearly three decades, PharmaEssentia said in a press release. ET is a rare blood disorder that causes the bone marrow to make too many platelets, which increases the risk for blood clots and bleeding.
The long-acting interferon-based therapy is designed to provide durable disease control and is indicated for use regardless of disease genotype or status, including in newly diagnosed patients who are naive to cytoreductive therapy. Ropeginterferon already carries an indication for polycythemia vera.
Approval was based on the open-label SURPASS ET trial involving 174 adults with ET who had an inadequate response or intolerance to hydroxyurea; 91 patients were randomly assigned to receive ropeginterferon, and 83 patients were randomly assigned to receive anagrelide. Patients in both arms also received low-dose aspirin unless contraindicated.
- FDA approved ropeginterferon alfa-2b for adult ET.
- First new ET therapy approval in nearly 30 years.
- Indicated regardless of genotype; includes newly diagnosed, cytoreductive-naive adults.
- SURPASS ET: 37.4% response vs 3.6% with anagrelide at months 9-12.
- Common AEs: transaminase ↑, anemia, fever, infection, pruritus, weight loss; boxed neuropsychiatric/autoimmune/ischemic/infectious risks.
Overall, 37.4% vs 3.6% of patients responded to ropeginterferon vs anagrelide at months 9 and 12. The response was defined by the modified European LeukemiaNet criteria and included blood count remission, improvement or nonprogression of splenomegaly, and the absence of bleeding or clotting events.
The most common adverse reactions with ropeginterferon were transaminase elevations, anemia, fever, bacterial infection, pruritus, and weight loss.
Labeling carries a black box warning of neuropsychiatric, autoimmune, ischemic, and infectious disorders.
The recommended starting dosage is 250 µg by subcutaneous injection. The dosage should be increased to 350 µg at 2 weeks and then to the maintenance dosage of 500 µg at 4 weeks. The maintenance dose should be given every 2 weeks unless a dose modification is required due to tolerability.
M. Alexander Otto is a physician assistant and award-winning journalist. He is also an MIT science journalism fellow. Email: aotto@medscape.net
