Journal
EXPERT REVIEW OF HEMATOLOGY
Volume 14, Issue 7, Pages 633-644Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1080/17474086.2021.1935854
Keywords
Ferroportin inhibitor; hepcidin; iron overload; non-transfusion-dependent beta-thalassemia; transfusion-dependent beta-thalassemia; vamifeport; VIT-2763
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Funding
- Vifor Pharma AG
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In beta-thalassemia, excessive iron absorption and ineffective erythropoiesis are key issues for treatment. Vamifeport, the first oral ferroportin inhibitor, shows promise in regulating iron levels based on initial studies.
Introduction: In beta-thalassemia, imbalanced globin synthesis causes reduced red blood cell survival and ineffective erythropoiesis. Suppressed hepcidin levels increase ferroportin-mediated iron transport in enterocytes, causing increased iron absorption and potentially iron overload. Low hepcidin also stimulates ferroportin-mediated iron release from macrophages, increasing transferrin saturation (TSAT), potentially forming non-transferrin-bound iron, which can be toxic. Modulating the hepcidin-ferroportin axis is an attractive strategy to improve ineffective erythropoiesis and limit the potential tissue damage resulting from iron overload. There are no oral beta-thalassemia treatments that consistently ameliorate anemia and prevent iron overload. Areas covered: The preclinical and clinical development of vamifeport (VIT-2763), a novel ferroportin inhibitor, was reviewed. PubMed, EMBASE and ClinicalTrials.gov were searched using the search term 'VIT-2763'. Expert opinion: Vamifeport is the first oral ferroportin inhibitor in clinical development. In healthy volunteers, vamifeport had comparable safety to placebo, was well tolerated and rapidly decreased iron levels and reduced TSAT, consistent with observations in preclinical models. Data from ongoing/planned Phase II studies are critical to define its potential in beta-thalassemia and other conditions associated with iron overabsorption and/or ineffective erythropoiesis. If vamifeport potentially increases hemoglobin and reduces iron-related parameters, it could be a suitable treatment for non-transfusiondependent and transfusion-dependent beta-thalassemia.
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