Recent drug approvals for acute myeloid leukemia

Catherine Lai, Kimberley Doucette, Kelly Norsworthy, Catherine Lai, Kimberley Doucette, Kelly Norsworthy

Abstract

Acute myeloid leukemia (AML) is the most common form of acute leukemia in adults, with an incidence that increases with age, and a generally poor prognosis. The disease is clinically and genetically heterogeneous, and recent advances have improved our understanding of the cytogenetic abnormalities and molecular mutations, aiding in prognostication and risk stratification. Until recently, however, therapeutic options were mostly limited to cytotoxic chemotherapy. Since 2017, there has been an explosion of newly approved treatment options both nationally and internationally, with the majority of new drugs targeting specific gene mutations and/or pivotal cell survival pathways. In this review article, we will discuss these new agents approved for the treatment of AML within the last 2 years, and will outline the mechanistic features and clinical trials that led to their approvals.

Keywords: Acute myeloid leukemia; Newly approved drugs; Novel treatments.

Conflict of interest statement

CL has served on an advisory board for Agios, Daiichi-Sankyo, and Jazz Pharmaceuticals and as a speaker for Astellas and Jazz Pharmaceuticals. KD and KN declare that they have no competing interests.

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