The pathophysiology and pharmacology of hepcidin

• The hormone hepcidin regulates systemic iron homeostasis.
• Hepcidin deficiency or excess contributes to the pathogenesis of iron disorders.
• Hepcidin agonists and antagonists are being developed as novel therapeutics.

Inappropriate production of the iron-regulatory hormone hepcidin contributes to the pathogenesis of common iron disorders. Absolute or relative deficiency of hepcidin causes iron overload in hereditary hemochromatosis and iron-loading anemias. Elevated hepcidin causes iron restriction in inflammatory conditions including autoimmune disease, critical illness, some cancers, and chronic kidney disease. Multiple agents targeting hepcidin and its regulators are under development as novel therapeutics for iron disorders. This review summarizes hepcidin biology and discusses the current landscape for hepcidin-targeting therapeutic strategies.