New targeted therapeutics in current clinical use for relapsed or refractory AML
| Agent . | Target . | CR+CRh/CRi/CRp % (CR %) . | Median survival, mo . | Approved population . | FDA-approval status . | Reference for FDA-approved indication and support for use in the unapproved setting . |
|---|---|---|---|---|---|---|
| Gilteritinib | FLT3 | 34 (21.1) | 9.3 | FLT3-mutated R/R AML | 2017 | 6 |
| Enasidenib | IDH2 | 26.6 (20.2) | 9.3 | IDH2-mutated R/R AML | 2017 | 18 |
| Ivosidenib | IDH1 | 30.4 (21.6) | 8.8 | IDH1-mutated R/R or untreated AML | 2018 | 19 |
| Gemtuzumab ozogamicin | CD33 | 33 (26) | 8.4 | CD33+ untreated or R/R AML in adults or pediatric patients 2 y or older | 2017 | 101 |
| HMA/LoDAC + venetoclax | BCL-2 | 67 (54)16 ; 54 (26)17 (untreated) | 17.516 ; 10.117 (untreated) | Untreated AML in patients 75 y and older unfit for chemotherapy | 2018 | 16,17,68-74 * |
| Agent . | Target . | CR+CRh/CRi/CRp % (CR %) . | Median survival, mo . | Approved population . | FDA-approval status . | Reference for FDA-approved indication and support for use in the unapproved setting . |
|---|---|---|---|---|---|---|
| Gilteritinib | FLT3 | 34 (21.1) | 9.3 | FLT3-mutated R/R AML | 2017 | 6 |
| Enasidenib | IDH2 | 26.6 (20.2) | 9.3 | IDH2-mutated R/R AML | 2017 | 18 |
| Ivosidenib | IDH1 | 30.4 (21.6) | 8.8 | IDH1-mutated R/R or untreated AML | 2018 | 19 |
| Gemtuzumab ozogamicin | CD33 | 33 (26) | 8.4 | CD33+ untreated or R/R AML in adults or pediatric patients 2 y or older | 2017 | 101 |
| HMA/LoDAC + venetoclax | BCL-2 | 67 (54)16 ; 54 (26)17 (untreated) | 17.516 ; 10.117 (untreated) | Untreated AML in patients 75 y and older unfit for chemotherapy | 2018 | 16,17,68-74 * |
BCL-2, B-cell lymphoma 2; CRh, CR with partial hematological recovery; CRp, CR with incomplete platelet recovery; IDH, isocitrate dehydrogenase; R/R, relapsed or refractory. See Table 1 for expansion of other abbreviations.
Selected additional recent publications and abstracts.