Table 1 Summary of key clinical outcomes in trials leading to recent approvals for AML therapies.

Trial Therapy	Regulatory approval in the US and Australia	Target population	SOC therapy	CR/CRi rates (%)		Median OS (months)
Trial Therapy	Regulatory approval in the US and Australia	Target population	SOC therapy	Active	Control	Active	Control
Midostaurin*	FDA: 28-Apr-2017 TGA: 17-May-2018 PBS: 01-Dec-2018	FLT3 mutant AML Age 18–59 y	7 + 3	58.9	53.5	74.7	25.6
Gemtuzumab ozogamicin†	FDA: 01-Sep-2017 TGA: 09-Apr-2020 PBS: 01-Mar-2022	De novo AML Age 50–70 y	7 + 3	81.3	74.8	27.5	21.8
CPX-351	FDA: 03-Aug-2017 TGA: 03-Jun-2022 PBS: n/a	Secondary or therapy related AML Age 60–75 y	7 + 3	47.7	33.3	9.6	6.0
CC-486	FDA: 01-Sep-2020 TGA: 08-Apr-2022 PBS: 01-Sep-2023	AML in CR1 and not candidates for HCT Age ≥55 y	Nil	-	-	24.7	14.8
Venetoclax^§	FDA: 21-Nov-2018 TGA: 20-Apr-2020 PBS: 01-Dec-2021	Age ≥75 y or unfit	AZA	66.4	28.3	14.7	9.6
Venetoclax^§	FDA: 21-Nov-2018 TGA: n/a PBS: n/a	Age ≥75 y or unfit	LDAC	48	13	8.4	4.1
Ivosidenib	FDA: 25-May-2022 TGA: 06-Apr-2023 PBS: n/a	Age ≥75 y or unfit	AZA	54	16	24.0	7.9

*Included only CR achieved by 60 days.
^†Included CR and CRp; median OS as per updated analysis (Lambert, Haematologica 2019).
^§Venetoclax in combination with azacitidine, or decitabine, or LDAC was granted accelerated approval by FDA on 21-Nov-2018, followed by full approval on 16-Oct-2020. In Australia, the approval is only for use in combination with azacitidine.
AML acute myeloid leukemia, AZA azacitidine, CR complete remission, CR1 first remission, CRi CR with incomplete hematologic recovery, CRp CR with incomplete platelet recovery, HCT hematopoietic cell transplantation, LDAC low-dose cytarabine, n/a not available, OS overall survival, PBS Pharmaceutical Benefits Scheme, SOC standard of care, TGA Therapeutic Goods Administration, y years.

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