Investigational and not approved by the FDA. This page is educational information only. It is not medical advice and not a recommendation to use anything.

IndexedBuilt automatically from public databases on 2026-09-30. Not yet hand-reviewed by PLOBT.How this works →

Rusfertide

Investigational

Hepcidin mimetic peptide

Also known as: PTG-300, PTG-300FB, Hepcidin Agonist PTG-300, Hepcidin Mimetic PTG-300, PTG300, PTG 300

Evidence score

58/ 100

Moderate evidence

A real evidence base, with gaps

Regulatory standing. In clinical trials, not yet approved
Limited human data. Some human data exists, not conclusive
Registered human trials. 6 registered studies on ClinicalTrials.gov, most advanced Phase 3
1 cited source. Includes primary / regulatory sources

Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.

What it is

Rusfertide, also known as PTG-300, is an investigational synthetic peptide designed to mimic hepcidin, the body's main hormone for controlling iron. By acting like hepcidin, it is thought to limit iron absorption and release into the blood, which may reduce excess red blood cell production in conditions such as polycythemia vera.

What the evidence shows

Automatic summaryLimited human data

Rusfertide is not FDA-approved; it has been studied in 6 registered human trials, with the most advanced reaching Phase 3, and about 18 published papers. Human evidence is growing, but its efficacy and safety have not been established by regulators.

Key facts

Molecular formula

C114H181N27O28S2

Molecular weight

2442 g/mol

Registered human trials

Live from ClinicalTrials.gov, refreshed daily.

6registered studies

Phase 3most advanced phase

2completed (sample)

1recruiting (sample)

PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit

Phase 2CompletedNCT04767802

A Study of Rusfertide in Adults With Polycythemia Vera in China

Phase 2Not Yet RecruitingNCT07765602

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Phase 3Active Not RecruitingNCT05210790

Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera

Phase 3Active Not RecruitingNCT06033586

Published literature

Live from Europe PMC. This is how much has been published, not how much is proven in humans.

21papers referencing Rusfertide

Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.

N Engl J Med2024

Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial.

Lancet Gastroenterol Hepatol2023

Hepcidin mimetics in polycythemia vera: resolving the irony of iron deficiency and erythrocytosis.

Curr Opin Hematol2023

Pharmacokinetics, pharmacodynamics, and tolerability of an aqueous formulation of rusfertide (PTG-300), a hepcidin mimetic, in healthy volunteers: A double-blind first-in-human study.

Eur J Haematol2024

Sources

Risks, side effects and study doses are only published on hand-reviewed profiles. Want this one reviewed, or spotted something wrong? Tell us.