Takeda (NYSE:TAK) and Protagonist Therapeutics (NASDAQ:PTGX) secured a major regulatory milestone on Monday as the U.S. Food and Drug Administration (FDA) granted Priority Review to rusfertide, an investigational treatment for polycythemia vera (PV).
The designation shortens the FDA’s review clock, with a Prescription Drug User Fee Act (PDUFA) goal date now set for the third quarter of 2026.
Rusfertide is a first-in-class hepcidin mimetic designed to regulate iron transition and control the overproduction of red blood cells.
In patients with PV, excessive red blood cell counts increase blood viscosity, leading to a high risk of life-threatening strokes and heart attacks.
The current standard of care often involves frequent phlebotomy (bloodletting) to keep hematocrit levels below 45%.
The regulatory filing is supported by the Phase 3 VERIFY study, where rusfertide met its primary and all key secondary endpoints.
Patients treated with the injection demonstrated superior hematocrit control and a significant reduction in the need for phlebotomies compared to those on standard care alone.