FDA Approves First-of-its-Kind Drug for Polycythemia Vera

The Food and Drug Administration recently approved rusfertide (Mimrylo, Takeda) as a new treatment for adults with polycythemia vera whose disease is not adequately controlled with existing therapies. Rusfertide is the first approved treatment for polycythemia vera that mimics hepcidin, a hormone that regulates iron availability in the body.

Rusfertide is administered subcutaneously once weekly and titrated to maintain hematocrit below 45%. By limiting the iron available for red blood cell production, the treatment is intended to reduce red blood cell overproduction.

FDA based its approval on the VERIFY trial, a multicenter, randomized, double-blind, placebo-controlled phase 3 study of 293 adults with polycythemia vera who required frequent phlebotomies despite standard-of-care therapy.

Between weeks 20 and 32, 76.9% of patients who received rusfertide required no phlebotomies, compared with 32.9% of patients who received placebo. The most common adverse reactions were injection site reactions and anemia.

Rusfertide received Priority Review.