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CFPS Research Archives

Rusfertide

Human clinical Hormonal Health 2 sources

Rusfertide is an investigational injectable hepcidin mimetic peptide designed to regulate iron metabolism. It is primarily researched for its ability to control red blood cell production in conditions like polycythemia vera by restricting iron availability.

01Dosing reference

Amount
10-80 mg
Frequency
Once or twice weekly
Cycle
Continuous management under clinical supervision
Reference figures, not a recommendationThese values reflect amounts described in the literature and vendor documentation this database indexes. Use the reconstitution calculator to convert them into syringe units.

02Mechanism of action

01

Ferroportin Binding

Rusfertide binds to ferroportin, the primary cellular iron exporter located on macrophages and enterocytes.

02

Receptor Internalization

The binding causes ferroportin to be internalized and degraded, preventing iron from being released into the bloodstream.

03

Reduced Erythropoiesis

Lower serum iron availability restricts the production of red blood cells, helping manage conditions characterized by excessive red blood cells.

03Human evidence

Rusfertide significantly reduced or eliminated the need for therapeutic phlebotomy in patients with polycythemia vera.

Phase 2 REVIVE study involving patients with polycythemia vera who required frequent phlebotomies.

The peptide was generally well tolerated, with injection site reactions being the most frequently reported adverse event.

Phase 2 clinical trials assessing safety and efficacy in patients with iron overload and polycythemia vera.

04Preclinical evidence

Rusfertide dose-dependently decreased serum iron levels and transferrin saturation.

Animal models of iron overload, including mice and rats.

Demonstrated a significantly prolonged half-life and improved stability compared to endogenous hepcidin.

Pharmacokinetic and pharmacodynamic studies in non-human primates.

05What is known vs. unknown

Reasonably established
  • Acts as a synthetic mimetic of the natural iron-regulating hormone hepcidin.
  • Effectively lowers serum iron and transferrin saturation by targeting ferroportin.
  • Reduces the need for phlebotomy in polycythemia vera patients.
  • Administered via subcutaneous injection, typically once or twice weekly.
Unknowns & limits
  • Long-term safety and efficacy beyond Phase 2 and Phase 3 trials are still being evaluated.
  • The long-term effects on bone marrow iron stores and potential for functional iron deficiency require further study.

06Safety & regulatory context

Regulatory statusRusfertide is currently an investigational drug and has received Orphan Drug and Breakthrough Therapy designations from the FDA for the treatment of polycythemia vera. It is not yet FDA-approved for general use. In clinical trials, it has been generally well tolerated, with the most common side effects being mild to moderate injection site reactions. Long-term safety data is still being collected.

07Compared with Endogenous Hepcidin

Rusfertide vs. Endogenous Hepcidin
Key difference
Rusfertide has a significantly longer half-life and improved stability compared to native hepcidin, allowing for practical therapeutic dosing.
When researchers discuss each
Hepcidin is discussed when explaining natural iron homeostasis, while Rusfertide is discussed as a therapeutic intervention for iron overload or excessive erythropoiesis.

08Glossary

Hepcidin
A hormone produced by the liver that serves as the master regulator of iron balance in the body.
Polycythemia Vera
A type of slow-growing blood cancer in which the bone marrow makes too many red blood cells.
Ferroportin
A transmembrane protein that transports iron from the inside of a cell to the outside, releasing it into the bloodstream.

09Knowledge check

Q1What is the primary mechanism of action of Rusfertide?
Q2Which condition is Rusfertide primarily being researched to treat?
Q3Which conclusion about Rusfertide's long-term use is best supported by the dossier?
Q4According to the dossier, what key difference distinguishes Rusfertide from endogenous hepcidin?
Q5Which statement accurately reflects Rusfertide's safety and regulatory context as described in the dossier?

10Sources

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Research and educational use onlyNothing on this site is medical advice, a prescription, or a recommendation for human use. Compounds documented here are research chemicals. Consult a qualified clinician before making any health decision.