Dr Mesa on the FDA Approval of Ropeginterferon Alfa-2b for Essential Thrombocythemia

[Hydroxyurea] lowers the platelets, but it doesn’t really change much about the disease. We have long felt that there is really an unmet need, and [we’re] excited about the great data with [ropeginterferon alfa-2b].

Ruben Mesa, MD, FACP, senior vice president of Atrium Health, as well as president and executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center, vice dean for Cancer Programs, and the Charles L. Spurr, MD, Professor of Internal Medicine at Wake Forest University School of Medicine, discussed the significance of the FDA approval of ropeginterferon alfa-2b-njft (Besremi) for adults with essential thrombocythemia (ET) and the longstanding unmet need the agent addresses in this myeloproliferative neoplasm (MPN).

On August 31, 2026, the FDA approved ropeginterferon alfa-2b for the treatment of adult patients with ET, regardless of genotype or disease status, including newly diagnosed patients naive to cytoreductive therapy. ET is one of the more common MPNs, with an estimated 150,000 to 200,000 people affected in the United States, many of whom can be younger patients, Mesa noted.

Despite the prevalence of ET, treatment options have historically been limited, according to Mesa. Until this approval, anagrelide (Agrylin) was the only FDA-approved therapy in the space, he explained. Hydroxyurea has been widely used largely on the basis of historic data, but it has never been formally approved for ET and carries a range of limitations, including effects on the skin and an increased risk of skin cancers, Mesa said. Fundamentally, hydroxyurea lowers platelet counts without meaningfully changing the underlying biology of the disease, he added.

The approval of ropeginterferon alfa-2b was supported by data from the phase 3 SURPASS-ET trial (NCT04285086). In patients with hydroxyurea-intolerant or -refractory ET, the durable modified European LeukemiaNet response rate at months 9 and 12 was 37.4% (95% CI, 27.4%-48.1%) with ropeginterferon alfa-2b (n = 91) vs 3.6% (95% CI, 0.8%-10.2%) with anagrelide (n = 83; difference, 33.9%; 95% CI, 23.5%-44.4%).

For Mesa, the approval addresses a need that has persisted across the MPN field, offering an option that may affect the biology of ET rather than control platelet counts alone. He expressed enthusiasm about the strength of the data supporting ropeginterferon alfa-2b and its potential to expand the treatment armamentarium for these patients.