FDA Approves First Hepcidin Mimetic for Polycythemia Vera

The FDA approved rusfertide (Mimrylo, Takeda)  for adults with polycythemia vera whose disease has not been adequately controlled with existing therapy. The first-in-class hepcidin mimetic is designed to limit the iron available for red blood cell production and reduce the need for repeated phlebotomy.

Polycythemia vera causes excessive red blood cell production, which can raise blood viscosity and increase the risk of thrombosis, stroke, and myocardial infarction. Treatment commonly aims to maintain hematocrit below 45%, often through phlebotomy in addition to other therapy.

The approval was supported by VERIFY, a multicenter, randomized, double-blind, placebo-controlled phase 3 trial involving 293 adults who continued to require frequent phlebotomies despite standard care. Participants received weekly subcutaneous rusfertide, beginning at 19 mg and titrated to keep hematocrit below 45%, or placebo for 32 weeks.

Between weeks 20 and 32, 76.9% of patients assigned to rusfertide did not meet criteria for phlebotomy, compared with 32.9% of those assigned to placebo. The most common adverse reactions were injection-site reactions and anemia.

The FDA granted the application priority review. Rusfertide offers a new mechanism for controlling erythrocytosis in patients who otherwise face a persistent disease-management burden.

Source: US Food and Drug Administration. News release. US Food and Drug Administration; August 28, 2026. Accessed August 30, 2026.