First-in-Class Drug Wins FDA Approval for Polycythemia Vera

The FDA has approved rusfertide (Mimrylo), a first-in-class hepcidin mimetic, for erythrocytosis in adults with polycythemia vera, a rare myeloproliferative neoplasm. The drug mimics hepcidin, the hormone regulating iron availability, with the aim of controlling haematocrit and reducing the need for therapeutic phlebotomy. In the earlier randomised phase 2 REVIVE trial (NEJM 2024, n=70), 60% of patients on rusfertide met the response criterion during the randomised withdrawal period versus 17% on placebo (p=0.002).