Library · Heart, blood & kidney · Treatment of polycythemia vera and iron disorders
Rusfertide
PTG-300, Rusfertide sodium
An investigational hepcidin-mimicking peptide for polycythemia vera, holding FDA Breakthrough Therapy, Orphan Drug, and Fast Track status. The Phase 3 VERIFY trial met its main goal by cutting the need for phlebotomy, with an NDA filing expected by the end of 2025.
Research score 90/100. Reference only, not a dose.
- Blood Pressure Regulation
- Cardiovascular Health
- Hormone Regulation
- Anti-inflammatory
- US status
- Investigational
- Approval
- Phase 3 Clinical Trial (Breakthrough Therapy Designation granted August 2025)
- Evidence
- Phase 3 VERIFY study met its primary endpoint for polycythemia vera. Positive Phase 2 REVIVE trial results also published.
- Research score
- 90 / 100
- Indication
- Investigational - Polycythemia Vera
- Origin
- USA
- Source category
- Heart & Cardiovascular
- Status group
- Investigational
Mechanism
Binds ferroportin, the cell's iron-export channel, triggering its internalization and breakdown via JAK/STAT-driven phosphorylation and ubiquitination, which lowers serum iron and limits excess red blood cell production.
Safety file
Generally well tolerated in trials; injection-site reactions are the most common adverse event.
- Injection site reactions
- Mild fatigue
- Transient headache
- Changes in iron parameters