Currently, midostaurin is the only Pharmaceutical Benefits Scheme (PBS)-listed targeted therapy approved to be used in combination with induction chemotherapy for newly diagnosed AML with a FLT3 internal tandem duplication (FLT3-ITD) or tyrosine kinase domain-activating mutation (FLT3-TKD) (DHS 2020; eviQ 2019b).

Enasidenib is a targeted oral therapy used as a single agent for treating refractory or relapsed IDH2-mutated AML. This drug has provisional TGA approval but is not PBS listed (February 2021). A phase 3 randomised trial of enasidenib or placebo in combination with induction chemotherapy for newly diagnosed IDH-mutated AML began in Australia in 2020. This trial also includes a randomisation of patients with IDH1-mutated AML to induction chemotherapy with or without the targeted IDH1 inhibitor ivosidenib.

Gilteritinib is a second-generation FLT3-inhibitor, approved by the TGA for patients with relapsed or refractory FLT3-mutant AML. This drug is not listed on the PBS (February 2021). It is administered as a single agent. A randomised trial of gilteritinib versus standard therapy (ADMIRAL) showed a survival benefit for patients receiving gilteritinib (Perl et al. 2019). A randomised trial of gilteritinib versus midostaurin in combination with induction chemotherapy in newly diagnosed FLT3-mutated AML began in Australia in 2020.

No immunotherapy drugs are TGA approved for treating AML.