The US Food and Drug Administration (FDA) has approved Rusfertide (Mimeylo), a hepcidin mimetic indicated for the treatment of erythrocytosis in adults with polycythemia vera (PV). Mimeylo is administered by subcutaneous injection. Mechanism of Action Rusfertide mimics the endogenous hormone hepcidin and blocks the iron transporter ferroportin. This reduces iron availability for red blood cell production, resulting in lower hematocrit levels.

Clinical Evidence Supporting Approval The efficacy of rusfertide was evaluated in the VERIFY Phase 3 clinical trial, involving 293 adults with polycythemia vera. Patients were randomized to rusfertide or placebo, with treatment continued through Week 32. Key findings included: 76.9% vs 32.9% achieved a response with rusfertide vs placebo Mean number of phlebotomies: 0.53 vs 1.82 62.6% vs 14.4% maintained hematocrit values below 45% Fatigue score…