Takeda has received FDA approval for MIMRYLO, or rusfertide, to treat erythrocytosis in adults with polycythemia vera, a chronic blood cancer characterized by overproduction of red blood cells.
MIMRYLO is a first-in-class, once-weekly subcutaneous hepcidin mimetic designed to regulate iron distribution and reduce the excess red blood cell production that drives elevated hematocrit in polycythemia vera. Maintaining hematocrit below 45% is the primary treatment goal because elevated levels can increase blood thickness and raise the risk of serious thrombotic events.
Takeda said approximately 90,000 people in the United States have polycythemia vera. The disease can lead to stroke, deep vein thrombosis and pulmonary embolism, and may cause severe fatigue, itching, difficulty concentrating and night sweats.
The company said an estimated 78% of patients experience uncontrolled hematocrit despite current standard treatment, including phlebotomy and cytoreductive therapies. Patients with uncontrolled hematocrit have a fourfold higher risk of cardiovascular death or major cardiovascular events, according to the announcement.
The approval was supported by the global Phase 3 VERIFY trial, which enrolled 293 patients with polycythemia vera. The study evaluated MIMRYLO plus current standard care against placebo plus current standard care in patients dependent on phlebotomy and experiencing uncontrolled hematocrit.
MIMRYLO met all efficacy endpoints and demonstrated a favorable safety profile, Takeda said. In the study, 76.9% of patients achieved a clinical response during Weeks 20 through 32. Response was defined as absence of phlebotomy eligibility.
Patients receiving MIMRYLO also showed hematocrit control, a reduced need for phlebotomy and improvement in fatigue measured by the PROMIS Fatigue Short Form 8a.
MIMRYLO was generally well tolerated through 52 weeks of treatment. The most common treatment-emergent adverse events were injection-site reactions and anemia. Injection-site reactions occurred in 56% of patients, while anemia occurred in 16%.
The treatment carries warnings for new or worsening thrombocytosis, injection-site reactions and embryo-fetal toxicity. Takeda said platelet counts should be monitored after starting MIMRYLO and during dose modifications.
The open-label extension of VERIFY remains ongoing, and Takeda plans to present additional findings at upcoming medical conferences. The company is also working with regulators outside the United States to potentially expand access globally.
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