Bivalirudin
An approved intravenous anticoagulant used during coronary intervention. Derived from leech saliva chemistry, with a very short and predictable action.
Cardiovascular & Blood
Rusfertide
An investigational hepcidin mimetic for polycythaemia vera. Phase 3 met its primary endpoint, substantially reducing the need for therapeutic phlebotomy.
Overview
Hepcidin is the hormone that controls how much iron enters the bloodstream. In polycythaemia vera the bone marrow overproduces red cells, and the standard management is repeated bloodletting to keep the blood from becoming too thick. Rusfertide mimics hepcidin, restricting the iron supply that red cell production depends on. Restrict the raw material and production falls — which means fewer phlebotomies.
A synthetic peptide mimetic of hepcidin that binds ferroportin, the sole cellular iron exporter, triggering its occlusion and internalisation. This traps iron in enterocytes, macrophages and hepatocytes, lowering transferrin saturation and restricting iron availability for erythropoiesis. In polycythaemia vera this constrains red cell mass independently of the underlying JAK2-driven clonal proliferation. The phase 3 VERIFY trial met its primary endpoint on clinical response defined by absence of phlebotomy eligibility, with phase 2 data showing sustained haematocrit control below 45%.
Research areas
Polycythaemia veraIron metabolismErythropoiesis
Dosing reported in the literature
Every protocol below is reproduced from a published study or approved labelling, with its source named. These are records of what was studied — not recommendations, and not a suggestion that any of them is appropriate for any person.
| Protocol as reported | Source |
|---|---|
| Weekly subcutaneous dosing titrated to maintain haematocrit below 45% without phlebotomy | VERIFY phase 3 protocol |
| Dose-titrated weekly administration over 28 weeks with phlebotomy requirement as the endpoint | Kremyanskaya et al., New England Journal of Medicine 2024 |
Trial status
Phase 3 VERIFY reported positive results in polycythaemia vera. Phase 2 completed with long-term extension data.
Reported safety signals
- Injection-site reactions are the most frequently reported adverse effect.
- Functional iron restriction is the mechanism, so iron-deficiency effects are expected rather than incidental.
- Long-term consequences of sustained pharmacological iron restriction are still being characterised.
- A cutaneous malignancy imbalance was noted in trial data and remains under evaluation.
Common questions
Why not just take iron away with diet?
Because the body recycles iron from old red cells far more than it absorbs from food — dietary restriction barely moves the available pool. Hepcidin controls both absorption and release from stores, which is why mimicking it works where diet does not.
Primary sources
Medical disclaimer
This page is for informational and research purposes only. Nothing here constitutes medical advice. The compounds discussed are research chemicals. Consult a qualified clinician before starting any protocol.
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