{"entity":{"id":"rusfertide","kind":"drug","name":"Rusfertide","aka":[],"tldr":"Rusfertide is the first drug to control polycythaemia vera's excess red cells by restricting iron, sparing patients repeated blood removal.","summary":"Rusfertide is an injectable hepcidin mimetic peptide that induces degradation of ferroportin, restricting iron supply to red-cell production and so holding haematocrit in range without repeated phlebotomy. It is intended for polycythaemia vera patients whose erythrocytosis persists despite standard therapy and who would otherwise depend on blood removal. In the VERIFY phase 3 trial (2025), significantly more patients on rusfertide plus standard therapy were phlebotomy-free versus placebo, with improved symptoms. The FDA approved it in August 2026 for erythrocytosis in adults with PV, the first drug to work through iron restriction in this disease; it was developed by Protagonist with Takeda. Long-term safety and whether this route reduces thrombosis as phlebotomy does remain open. In short, rusfertide treats the excess red cells by starving them of iron rather than draining them.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Rusfertide","links":[{"label":"FDA novel approvals 2026","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera"],"sections":[],"technologies":["peptide-drug-conjugate"],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["verify","nct07648030","nct06033586"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mimrylo","modality":"Hepcidin mimetic peptide","mechanism":"Injectable hepcidin mimetic that degrades ferroportin, restricting iron availability to erythropoiesis and controlling haematocrit without phlebotomy.","approvals":[{"region":"US","year":2026,"indication":"Erythrocytosis in adults with polycythaemia vera"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/rusfertide/","neighbours":{"cancer":[{"id":"myeloproliferative-neoplasms","kind":"cancer","name":"Myeloproliferative neoplasms (PV, ET, myelofibrosis)","route":"/cancers/myeloproliferative-neoplasms/"},{"id":"polycythaemia-vera","kind":"cancer","name":"Polycythaemia vera (PV)","route":"/cancers/polycythaemia-vera/"}],"technology":[{"id":"peptide-drug-conjugate","kind":"technology","name":"Peptide-drug & small-molecule-drug conjugates","route":"/technologies/peptide-drug-conjugate/"}],"company":[{"id":"protagonist-therapeutics","kind":"company","name":"Protagonist Therapeutics","route":"/companies/protagonist-therapeutics/"},{"id":"takeda","kind":"company","name":"Takeda","route":"/companies/takeda/"}],"trial":[{"id":"nct07648030","kind":"trial","name":"A Study of Rusfertide in Japanese Adults With Polycythemia Vera","route":"/trials/nct07648030/"},{"id":"nct06033586","kind":"trial","name":"Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera","route":"/trials/nct06033586/"},{"id":"verify","kind":"trial","name":"VERIFY","route":"/trials/verify/"}],"term":[{"id":"hepcidin","kind":"term","name":"Hepcidin","route":"/terms/hepcidin/"},{"id":"phlebotomy","kind":"term","name":"Phlebotomy (venesection)","route":"/terms/phlebotomy/"}],"idea":[{"id":"idea-pv-hepcidin-first","kind":"idea","name":"Hepcidin-based control as first-line treatment in low-risk polycythaemia vera","route":"/ideas/idea-pv-hepcidin-first/"}]}}