Rusfertide

DB17724ApprovedInvestigationalProtein Based Therapies

Rusfertide is a first-in-class hepcidin mimetic indicated for erythrocytosis in adults with polycythemia vera, acting through ferroportin blockade to limit iron availability for red blood cell production. It is a synthetic cyclic peptide that blocks the iron transporter ferroportin, reducing the availability of iron for red blood cell production and lowering hematocrit.

Mechanism: Rusfertide is a mimetic of the endogenous hormone hepcidin that blocks the iron transporter ferroportin. Inhibition of ferroportin by rusfertide reduces the availability of iron for production of red blood cells, resulting in lower hematocrit levels.
Curator reviewed · 2 references
Primary indication: Rusfertide is indicated for the treatment of erythrocytosis in adults with polycythemia vera.Curator reviewed · 1 structured indication
First approval: United States, 2026
Code names: PTG-300 · PTG-300FB · TAK-121
Brand names: Mimrylo

Resolves to

Compound

UNIIXM71MYX0IQCAS1628323-80-7

Clinical / RWD

RxNorm2751595

Targets

Q9NP59SLC40A1

What you can answer from here — as of October 29, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

8Clinical trialsPhase, status and sponsor resolved per trial

1Structured indicationCondition, population, route and combination as fields, not prose

5Marketed productsAcross 1 country and 1 labeller

3+ReferencesStructured and connected to the statements they support

Which drugs share a target with Rusfertide, and which of those have an active Phase 3 trial?

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