Table 2:
Targeted AML Therapies.
| Name | Mechanism of action | Target cell type | Approved for… | Combined with… | Median OS |
|---|---|---|---|---|---|
| Midostaurin (Rydapt) [105] | Inhibitor of FLT3, PKC, serine/ threonine + tyrosine kinases | Blasts + LSCs | De novo FLT3-mutated AML | 7+3 induction, HiDAC consolidation, azacitidine | With 7+3 = 74.7 mo. |
| Gilteritinib (Xospata) [106] | Inhibitor of FLT3 | Blasts + LSCs | R/R FLT3-mutated AML | Single agent | 9.3 mo. |
| Ivosidenib (Tibsovo) [107], [108] | Inhibitor of IDH1 | Blasts + LSCs | De novo AML in patients >75 years or R/R IDH1 mutated AML | Single agent |
De novo = 8.8 mo. R/R - 12.6 mo. |
| Enasidenib (Idhifa) [109] | Inhibitor of IDH2, induces terminal differentiation | Blasts | R/R IDH2-mutated AML | Single agent | 8.8 mo. |
| Gemtuzumab ozogamicin (Mylotarg) [110] | Anti-CD33 monoclonal humanized antibody | Blasts + CMPs | De novo or R/R CD33+ AML | Daunorubicin or cytarabine, or single agent | With dau/Ara-C = 17.3 mo. Single agent = 4.9 mo. |
| Venetoclax (Venclexta) [111] | Inhibitor of BCL-2 | Blasts + LSCs | De novo AML in patients >75 years or with comorbidities | Azacitidine | 17.5 mo. |
| Glasdegib (Daurismo) [112] | Inhibitor of the Hedgehog pathway | Blasts + LSCs | De novo AML in patients >75 years or with comorbidities | Low-dose cytarabine (LDAC) | 8.8 mo. |