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Commentary|Videos|March 14, 2026

Rusfertide May Become Part of Polycythemia Vera Armamentarium

Treatment with rusfertide appears to reliably control hematocrit among those with polycythemia vera, according to Andrew Kuykendall, MD.

In March 2026, the FDA granted priority review to rusfertide as a potential treatment option for patients with polycythemia vera. Supporting findings for the new drug application primarily came from the phase 3 VERIFY trial (NCT05210790), in which rusfertide plus standard-of-care (SOC) therapy improved responses and other outcomes vs SOC alone.

Following this regulatory milestone, CancerNetwork® spoke with Andrew Kuykendall, MD, an investigator on the VERIFY trial, about how the potential FDA approval of rusfertide may impact the treatment paradigm for those with polycythemia vera. Kuykendall, an associate member in the Department of Malignant Hematology at Moffitt Cancer Center, stated that rusfertide may become part of the treatment armamentarium for this disease.

Specifically, Kuykendall noted that rusfertide can reliably control hematocrit among patients, which clinicians in the field have emphasized as a core tenant of polycythemia vera management. Additionally, he said the agent appears to have a manageable safety profile.

Transcript:

Rusfertide is going to be part of our armamentarium going forward for polycythemia vera. This is an agent that is extremely well tolerated; that’s probably to be decided by the patients, but it certainly seems like that. It certainly [has a] very manageable safety profile, and it’s extremely effective across the board. It does seem like it’s able to get reliable control of hematocrit, and this is something that we’ve said for a long time as a core tenet of [treating patients with] polycythemia vera: to achieve stable and optimal control of hematocrit to reduce the risk for thrombotic events. This is something that could be a part of every patient with polycythemia vera’s treatment history, maybe not forever, but at least at some point during their disease. It’s something that can be combined with other therapies that can be used for their disease too. It’s something that we’re going to be going to be talking about a lot with patients.

Reference

Takeda and Protagonist announce U.S. Food and Drug Administration accepts new drug application and grants priority review for rusfertide as a potential first-in-class therapy for polycythemia vera. News release. Takeda. March 2, 2026. Accessed March 13, 2026. https://tinyurl.com/3njzamwp

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