New drugs for the treatment of microcytic anemia
| Drug . | Mechanism/effect . | Preclinical data/clinical trial . |
|---|---|---|
| Hepcidin analogs and mini-hepcidin | Replace endogenous hepcidin | Clinical trial |
| Anti-TMPRSS6 (ASO, siRNA) | Hepcidin inhibition | Clinical trial |
| FPN inhibitor VIT-2763 | Blocking the hepcidin receptor | Clinical trial |
| Transferrin injection | Decreasing transferrin receptor/reducing iron uptake | Preclinical study in animal model |
| Luspatercept | Activin receptor IIB ligand trap/ameliorating anemia and reducing hepcidin inhibition | Clinical trial |
| Protoporphyrin IX | Reducing iron cycling by inhibiting heme oxygenase 1 | Preclinical study in animal model |
| Anti–IL-6 and anti–IL-6R | Reducing the hepcidin signaling pathway | Preclinical study in animal model |
| Anti-BMP6 MoAb | Reducing the hepcidin signaling pathway | Clinical trial |
| BMP receptor inhibitors | Reducing the hepcidin signaling pathway | Preclinical study in animal model |
| Antihemojuvelin MoAb | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
| Antihepcidin MoAb | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
| Antihepcidin Spiegelmer | Reduced hepcidin/correction of hypoferremia/correction of anemia | Clinical trial |
| Antihepcidin anticalin | Reduced hepcidin/correction of hypoferremia/correction of anemia | Clinical trial |
| Antiferroportin MoAb GDP | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
| Drug . | Mechanism/effect . | Preclinical data/clinical trial . |
|---|---|---|
| Hepcidin analogs and mini-hepcidin | Replace endogenous hepcidin | Clinical trial |
| Anti-TMPRSS6 (ASO, siRNA) | Hepcidin inhibition | Clinical trial |
| FPN inhibitor VIT-2763 | Blocking the hepcidin receptor | Clinical trial |
| Transferrin injection | Decreasing transferrin receptor/reducing iron uptake | Preclinical study in animal model |
| Luspatercept | Activin receptor IIB ligand trap/ameliorating anemia and reducing hepcidin inhibition | Clinical trial |
| Protoporphyrin IX | Reducing iron cycling by inhibiting heme oxygenase 1 | Preclinical study in animal model |
| Anti–IL-6 and anti–IL-6R | Reducing the hepcidin signaling pathway | Preclinical study in animal model |
| Anti-BMP6 MoAb | Reducing the hepcidin signaling pathway | Clinical trial |
| BMP receptor inhibitors | Reducing the hepcidin signaling pathway | Preclinical study in animal model |
| Antihemojuvelin MoAb | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
| Antihepcidin MoAb | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
| Antihepcidin Spiegelmer | Reduced hepcidin/correction of hypoferremia/correction of anemia | Clinical trial |
| Antihepcidin anticalin | Reduced hepcidin/correction of hypoferremia/correction of anemia | Clinical trial |
| Antiferroportin MoAb GDP | Reduced hepcidin/correction of hypoferremia/correction of anemia | Preclinical study in animal model |
ASO, antisense specific oligonucleotides; BMP, bone morphogenetic protein; FPN, ferroportin; GDP, guanosine 5′-diphosphate encapsulated in lipid vesicle; IL, interleukin; MoAb, monoclonal antibodies; siRNA, short interfering RNA; VIT-2763, small molecule oral ferroportin inhibitor.