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News
Rusfertide (Mimrylo) has reached a new milestone in polycythemia vera treatment. Updated NCCN guidelines now designate it as a Category 1 option for both low- and high-risk disease — the only therapy to hold that distinction across both groups. The recommendation follows its FDA approval in August 2026 and strong phase 3 VERIFY trial data showing a 76.9% clinical response rate vs. 32.9% with placebo.
Rusfertide (Mimrylo, Takeda) has earned a Category 1 designation in the updated NCCN Clinical Practice Guidelines for Myeloproliferative Neoplasms — and it's the only therapy to hold that status across both low- and high-risk polycythemia vera. The update follows the drug's FDA approval in August 2026, cementing its place as a new option for patients whose disease hasn't been adequately controlled with existing therapies.
The recommendation is backed by two key trials: the phase 3 VERIFY trial and the phase 2 REVIVE trial. In VERIFY, rusfertide plus standard of care delivered a 76.9% clinical response rate compared to just 32.9% with placebo — a notable difference that highlights how many patients currently fall short of adequate hematocrit control. Rusfertide works as a first-in-class hepcidin-mimicking peptide, regulating iron homeostasis to reduce red blood cell overproduction, and is self-administered via weekly subcutaneous injection.
By the Numbers
Why it matters: Polycythemia vera patients face serious risks — stroke, DVT, pulmonary embolism — from uncontrolled red blood cell overproduction. With many patients failing to achieve hematocrit control on current therapies, rusfertide's Category 1 NCCN status gives clinicians a well-evidenced new tool to address that gap.
