The U.S. Food and Drug Administration has granted Priority Review to the New Drug Application for rusfertide, a potential new treatment for adults with polycythemia vera (PV). A final approval decision is expected in the third quarter of 2026. PV causes the body to produce too many red blood cells, which thickens the blood and raises the risk of dangerous clots. Many patients currently need regular blood draws (phlebotomy) to control the condition.
Rusfertide is a once-weekly injection that mimics hepcidin, the body’s natural hormone that controls iron levels. By limiting iron, it reduces red blood cell production and helps control the disease at its source. In the Phase 3 VERIFY trial, about 77% of patients receiving rusfertide achieved a clinical response, compared with about 33% in the placebo group. Patients on rusfertide needed fewer phlebotomies and were more likely to keep their hematocrit levels below 45%. Many also reported less fatigue and improved symptoms, and most responders maintained benefits at one year.
The drug was generally well tolerated. The most common side effects were mild injection-site reactions, anemia, and fatigue. If approved, rusfertide could reduce or even eliminate the need for frequent blood draws and offer a new treatment option for people living with polycythemia vera.