Rusfertide Approved for Polycythemia Vera
Key Insights
The FDA has approved rusfertide (Mimrylo) for adults with polycythemia vera (search), a rare blood cancer marked by excessive blood cell production.
Rusfertide is a first-in-class hepcidin mimetic that blocks iron release into the bloodstream, limiting iron available to the bone marrow for red blood cell production.
Approval was based on the phase III VERIFY trial, where 76.9% of rusfertide-treated patients maintained safe red blood cell levels without phlebotomy versus 32.9% on placebo.
The U.S. Food and Drug Administration (search) has approved rusfertide (Mimrylo) for adult patients with polycythemia vera (search), a rare, slow-growing blood cancer characterized by excessive blood cell production. Rusfertide is a first-in-class agent that mimics the natural hormone hepcidin, which regulates iron absorption, storage, and distribution in the body.
By blocking the release of iron into the bloodstream, rusfertide reduces the iron available to the bone marrow for red blood cell production, helping keep the percentage of red blood cells within a safe limit. The approval was supported by the VERIFY multicenter, randomized, double-blind phase III trial, which enrolled 293 adults with polycythemia vera (search) who required frequent phlebotomies to control red blood cell levels. Participants were randomly assigned 1:1 to 19 mg rusfertide or placebo for 32 weeks alongside their ongoing polycythemia vera treatment.
During follow-up, 76.9% of patients in the rusfertide arm maintained safe red blood cell levels without phlebotomy, compared with 32.9% in the placebo arm. The recommended starting dose is 19 mg once weekly by subcutaneous administration, adjustable based on efficacy or safety to a maximum of 108 mg per week. Polycythemia vera (search) is a myeloproliferative neoplasm in which excess red blood cells thicken the blood and increase clot risk, potentially leading to strokes or heart attacks; an estimated 65,000 people in the United States live with the disease, with median survival of 14 to 27 years.