Table 3.
Drug | Type | Mechanism of Action | Mode of Administration | Response Rate | References |
---|---|---|---|---|---|
Used in TA-TMA | |||||
Eculizumab | mAb | Inhibition of C5 and C5b9 formation | intravenous | Children 67–78%Adults 33–60% | [25,50,51,52,53,54,55,56,57,58,59] |
Ravulizumab | mAb | Inhibition of C5 and C5b9 formation | intravenous | Under evaluation | NCT04543591 |
Coversin | Small protein | Inhibition of C5 and C5b9 formation | subcutaneous | Single case | [74] |
Narsoplimab | mAb | MASP-2 inhibition | intravenous | Adults 61% | [79] NCT02222545 |
Pegcetacoplan | Peptide | C3 inhibition | subcutaneous | Under evaluation | NCT05148299 |
Never used in TA-TMA | |||||
Crovalimab | mAb | Inhibition of C5 and C5b9 formation | subcutaneous | ||
Iptacopam | Small molecule | Factor B inhibition | oral | ||
Danicopan | Small molecule | Factor D inhibition | oral | ||
BCX9930 | Small molecule | Factor D inhibition | oral | ||
Avacopan | Small molecule | C5a inhibition | oral | ||
AMY-101 | Peptide | C3 inhibition | subcutaneous |