Table 1

New agents for AML with recent U.S. FDA approval.

	Molecular target	Indication (setting)		Representative trials	OS benefit	FDA approval	In Korea (Jan. 2020)

		New	Rel/ref
Midostaurin	FLT3 ITD/TKD	O (combia))		Phase 3 RATIFY	Yes	Apr. 2017	Available
Gilteritinib	FLT3 ITD/TKD		O (single)	Phase 3 ADMIRAL	Yes	Nov. 2018	Not available
Gemtuzumab ozogamicin (GO)	CD33+	O (combi or single)	O (combi or single)	Phase 3 ALFA-0701	No (but benefit in EFS)	Jul. 2017	Available (via KOEDC)
				Phase 3 AML-19	Yes
				Phase 2 MyloFrance-1	NA
CPX-351	t-AML	O		Phase 3 (NCT01696084)	Yes	Aug. 2017	Not available
CPX-351	AML-MRC	O		Phase 3 (NCT01696084)	Yes	Aug. 2017	Not available
Enasidenib	IDH2		O (single)	Phase 1/2 study (NCT01915498)	NA	Aug. 2017	Not available
Ivosidenib	IDH1		O (single)	Phase 1 dose-escalation/dose-expansion study (NCT02074839)	NA	Jul. 2018	Not available
Venetoclax	BCL2	O (combib))		Phase 1 dose-escalation study (NCT02203773)	NA	Nov. 2018	Available
Venetoclax	BCL2	O (combib))		Phase 1/2 study (NCT02287233)	NA	Nov. 2018	Available
Glasdegib	Hedgehog signaling pathway		O (combic))	Phase 2 (NCT01546038)	Yes	Nov. 2018	Not available

^a)Midostaurin in combination with daunorubicin and cytarabine induction and cytarabine consolidation.

^b)Hypomethylating agents (HMA) such as decitabine or azacitidine with venetoclax (400 mg); low dose Ara-C (LDAC) with venetoclax (600 mg).

^c)Glasdegib and LDAC.

Abbreviations: BCL2, B-cell lymphoma 2; EFS, event-free survival; FLT3, FMS-like tyrosine kinase 3; IDH, isocitrate dehydrogenase; ITD, internal tandem duplication; KOEDC, Korea Orphan & Essential Drug Center; NA, not assessable; OS, overall survival; rel/Ref, relapsed or refractory; t-AML, therapy-related AML; AML-MRC, AML with myelodysplasia related changes; TKD, tyrosine kinase domain.

Blood Res 2020;55:S14~S18 https://doi.org/10.5045/br.2020.S003