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.
Notably, the drug also showed improvement in patient-reported symptoms, such as fatigue.
The partnership, formed in early 2024, grants Takeda exclusive worldwide rights to the drug, while Protagonist retains a 50/50 profit-share option in the U.S. market.
Rusfertide has already received Breakthrough Therapy and Orphan Drug designations, reflecting the high unmet need for PV treatments that can provide consistent systemic control without the burden of chronic blood draws.