Drug intelligence / Profile preview

vamifeport

Development stage
Phase 2
Lead developer
CSL Vifor
Modality
Small Molecules
Administration
Oral
01

Overview

**Vamifeport** is a first-in-class, orally administered small molecule inhibitor of ferroportin (SLC40A1), the only known cellular iron exporter. By inhibiting ferroportin, vamifeport reduces iron efflux from cells such as macrophages and enterocytes, thereby decreasing plasma iron levels and limiting non-transferrin-bound iron (NTBI) formation. This mechanism is particularly relevant in conditions characterized by ineffective erythropoiesis and dysregulated iron homeostasis, such as beta-thalassemia and sickle cell disease. Preclinical studies have shown that vamifeport ameliorates anemia, improves erythropoiesis, reduces NTBI after transfusion, and prevents excessive intestinal iron absorption. Clinical trials are ongoing for beta-thalassemia, sickle cell disease, and iron overload disorders[1][2][4][5][7].

Other names
vamifeport hydrochlorideVIT-2763 3HClVIT2763 3HClVIT 2763 3HClVIT-2763 trihydrochlorideVIT2763 trihydrochlorideVIT 2763 trihydrochlorideVamifeport [INN]Vamifeport [USAN]
02

Targets

SLC40A1 (Ferroportin)

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