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Hepcidin (specifically hepcidin-25) is a synthetic equivalent of the endogenous 25-amino acid peptide hormone hepcidin, which acts as the master regulator of systemic iron homeostasis. Developed by La Jolla Pharmaceutical Company (as LJPC-401), hepcidin binds directly to the cellular iron exporter ferroportin (SLC40A1), inducing its internalization and lysosomal degradation. This action limits iron efflux from macrophages and enterocytes into the circulation, thereby reducing serum iron levels. LJPC-401 was investigated in Phase 2 clinical trials for the treatment of iron overload disorders, including hereditary hemochromatosis and beta thalassemia. However, due to a lack of efficacy in a pivotal Phase 2 trial for myocardial iron overload in beta thalassemia, its development was discontinued in late 2019.
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