Rusfertide - (REVIVE)

March 1, 2026: Takeda (TAK) and Protagonist Therapeutics (PTGX) announced the FDA accepted the New Drug Application (NDA) for rusfertide, an investigational first-in-class hepcidin mimetic peptide for treatment of adults with polycythemia vera (PV), and granted Priority Review. The FDA set a PDUFA target action date in the third quarter of this calendar year. Rusfertide has also received Breakthrough Therapy, Orphan Drug, and Fast Track designations.

The NDA is primarily based on Phase 3 VERIFY (NCT05210790) positive 32-week primary analysis and 52-week results, plus four-year efficacy/safety data from Phase 2 REVIVE (NCT04057040) and long-term extension THRIVE (NCT06033586). In VERIFY, rusfertide met the primary endpoint and all four key secondary endpoints, with higher response rates vs standard of care (rusfertide + standard of care vs placebo + standard of care), including hematocrit control, reduced phlebotomy requirements, and improved patient-reported fatigue and symptom burden. Through 52 weeks, the most common AEs were injection site reactions (47.4%), anemia (25.6%), and fatigue (19.6%); serious AEs occurred in 8.1% of rusfertide-treated patients.

The release also notes a January 2024 worldwide license/collaboration agreement between Protagonist and Takeda for rusfertide and that Protagonist has an option to co-commercialize in the U.S. under a 50/50 profit-and-loss structure.