- FDA set a PDUFA target action date for rusfertide in Q3 2026.
- Rusfertide is a hepcidin mimetic peptide for polycythemia vera.
- The NDA is based on Phase 3 VERIFY and Phase 2 REVIVE/THRIVE studies.
- Rusfertide showed improved hematocrit control and reduced phlebotomy needs.
FDA Review and Designations
The FDA has accepted the New Drug Application (NDA) for rusfertide, granting it Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date is set for the third quarter of 2026. Rusfertide has also received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA.
Rusfertide's Clinical Efficacy
Rusfertide, a hepcidin mimetic peptide, is designed to treat polycythemia vera (PV), a condition characterized by excessive red blood cell production. The primary treatment goal in PV is to maintain hematocrit levels below 45% to prevent thrombotic events. In clinical studies, rusfertide demonstrated significant improvements in hematocrit control, reduced phlebotomy requirements, and improved patient-reported outcomes.
Clinical Study Data
The NDA submission is primarily based on the Phase 3 VERIFY study and long-term data from the Phase 2 REVIVE and THRIVE studies. In the VERIFY study, rusfertide met its primary endpoint and all key secondary endpoints, showing higher response rates compared to the standard of care. The treatment was generally well-tolerated, with common adverse events being injection site reactions, anemia, and fatigue.
Collaboration and Development
Protagonist Therapeutics discovered rusfertide and led its development through Phase 3 studies. In January 2024, Protagonist and Takeda entered a worldwide license and collaboration agreement, with Takeda responsible for the U.S. regulatory strategy and future global filings. Protagonist retains an option to co-commercialize in the U.S. or opt-out, granting Takeda a worldwide license.
