Acceptance of New Drug Application by the U.S. Food and Drug Administration for rusfertide, a Potential First-in-Class Treatment for Polycythemia Vera, in Collaboration with Protagonist Therapeutics, and Its Designation for Priority Review

Overview of New Drug Application

Takeda Pharmaceutical Company Limited announced that the U.S. Food and Drug Administration (FDA) accepted the new drug application (NDA) for rusfertide, co-developed with Protagonist Therapeutics, on February 27, 2026, and designated it for priority review. Rusfertide is a first-in-class treatment for polycythemia vera (PV), with a PDUFA target action date set for Q3 2026 (July to September). Rusfertide is a hepcidin peptide mimetic, which, when combined with standard treatment, has demonstrated more than double the clinical response rate, significantly improving hematocrit control and reducing phlebotomy frequency.

Future Outlook and Partnership

In January 2024, Takeda entered into a global license and collaboration agreement with Protagonist Therapeutics for rusfertide and is leading the strategic implementation of the NDA and future regulatory submissions in the U.S. Protagonist Therapeutics holds co-promotion rights in the U.S. on a 50:50 profit-sharing basis and retains opt-out rights. Rusfertide is administered via once-weekly subcutaneous self-injections and has shown generally favorable tolerability in clinical trials. It has also received FDA designations for Breakthrough Therapy, Orphan Drug, and Fast Track, with approval anticipated within 2026.