Announcement of Long-term rusfertide Data Demonstrating Sustained Hematocrit Control in Polycythemia Vera at the 67th Annual Meeting of the American Society of Hematology (ASH) in Collaboration with Protagonist Therapeutics

Product and Service Overview

Rusfertide is a novel therapeutic agent that demonstrated sustained hematocrit control and clinical efficacy over 52 weeks in the Phase 3 VERIFY trial for patients with polycythemia vera (PV). Administered once weekly by self-injection in addition to standard therapy, 61.99% of patients maintained a clinical response without requiring phlebotomy. The safety profile was favorable, with local injection site reactions, anemia, and fatigue being the main adverse events. Currently, rusfertide has received Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation from the FDA, and its impact on fiscal year ending March 2026 earnings is expected to be minor.

Market Impact and Outlook

Rusfertide presents a potential new standard of care for PV patients struggling to control hematocrit levels with existing treatments. The long-term extension trial THRIVE confirmed sustained efficacy and safety, along with a significant reduction in the annual thrombosis rate. Takeda, in collaboration with Protagonist Therapeutics, is preparing a marketing authorization application to the U.S. FDA, expected to expand treatment options and improve quality of life for PV patients. This will strengthen Takeda’s competitiveness in the oncology field and represents a strategically important business development.