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Articles / Hematology 08/29/2026

Rusfertide Approval Offers Novel Treatment Approach for Patients With PV

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On August 28, 2026, the FDA approved rusfertide (MIMRYLO) for the treatment of adults with polycythemia vera (PV), according to a press release from Takeda, the manufacturer of the drug. 

PV is characterized by overproduction of red blood cells leading to an increased risk of cardiovascular events. Even with treatment, many patients with PV are dependent on regular blood draws to reduce their hematocrit level, which is the proportion of red blood cells in blood. Rusfertide is a first-in-class hepcidin mimetic that regulates iron in the body to limit the overproduction of red blood cells and reduce hematocrit levels, the release stated.

The approval of rusfertide was based on the phase 3 VERIFY trial, which compared rusfertide with placebo in patients with polycythemia vera dependent on phlebotomies despite ongoing standard therapy. The primary endpoint was the proportion of patients who stopped meeting criteria for phlebotomies and did not receive phlebotomies in weeks 20 to 32 of treatment. Overall, 76.9% of patients in the rusfertide group versus 32.9% of patients in the placebo group achieved the primary endpoint. The prescribing information for rusfertide described additional efficacy endpoints from VERIFY:

Efficacy results of VERIFY study comparing placebo and MIMYROLO in cancer patients, highlighting response rates, phlebotomy, and HCT values.
Summary of VERIFY study showing significant differences in response, phlebotomy, and HCT between placebo and MIMYROLO treatments.

The most common treatment-emergent adverse events (AEs) with rusfertide were injection site reactions, anemia, and fatigue. Specific all-grade and grade 3 rates for each arm in VERIFY were as follows: 

Table showing adverse reactions like injection site reactions, anemia, thrombocytosis, and dyspnea in patients from MIMYRO and placebo groups in a clinical trial.
Clinical trial data comparing adverse reactions such as injection site reactions, anemia, thrombocytosis, and dyspnea between MIMYRO and placebo groups.

“The approval of MIMRYLO offers clinicians and patients a novel, first-in-class therapy that targets erythrocytosis, which drives excess red blood cell production in PV,” said VERIFY lead investigator Andrew Kuykendall, MD, of Moffitt Cancer Center. “The strength and consistency of the VERIFY data give me real confidence in MIMRYLO’s potential to advance how we treat PV in everyday practice and to maintain hematocrit control.”