Takeda Receives U.S. FDA Approval of MIMRYLO (rusfertide), Marking a Potential Shift in the Treatment Paradigm for Polycythemia Vera

Takeda (TSE:4502/NYSE:TAK) announced U.S. Food and Drug Administration (FDA) approval of the New Drug Application (NDA)* for MIMRYLO (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV), a blood cancer.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260824773270/en/

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MIMRYLO is a first-in-class hepcidin mimetic designed to regulate iron distribution in the body and red blood cell overproduction to control hematocrit levels, which is the ratio of red blood cells to the total amount of blood in the body. Maintaining controlled hematocrit levels below 45% is the primary treatment goal in PV.¹

“For patients living with PV, uncontrolled hematocrit can have serious consequences, including an elevated risk of life-threatening thrombotic events," said Andrew T. Kuykendall, M.D., VERIFY lead investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center. “Current treatments, such as phlebotomy, leave a significant gap for too many patients and can pose challenges to daily life and routines. 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. 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.”

Uncontrolled Hematocrit is a Challenge in the Treatment of PV
Affecting approximately 90,000 people in the U.S., PV is characterized by the overproduction of red blood cells (erythrocytosis), leading to elevated hematocrit which can increase blood viscosity, or thickness.^2,3 This has the potential to result in life-threatening thrombotic events, including stroke, deep vein thrombosis and pulmonary embolism.⁴ Maintaining hematocrit levels consistently below 45% can prevent thrombotic events and alleviate burdensome symptoms, including severe fatigue, pruritus (itching), difficulty concentrating and night sweats.⁴ An estimated 78% of patients still experience uncontrolled hematocrit with current standard of care, including phlebotomy and cytoreductive therapies.⁵ Patients with PV experiencing uncontrolled hematocrit have a four times higher risk of cardiovascular death or major cardiovascular events.⁴

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