New Food and Drug Administration-Approved and Emerging Novel Treatment Options for Acute Myeloid Leukemia

Pharmacotherapy. 2018 Nov;38(11):1143-1154. doi: 10.1002/phar.2180. Epub 2018 Oct 17.

Abstract

Advancements in the treatment of acute myeloid leukemia (AML) have been sparse during the past several decades, and the disease continues to have a poor prognosis. However, in 2017 alone, four new medications approved by the U.S. Food and Drug Administration (FDA) for the treatment of AML reached the market. Midostaurin, liposomal cytarabine and daunorubicin, enasidenib, and gemtuzumab ogozamicin all showed benefit in respective clinical trials to gain approval for the treatment of AML in various patient populations. Additionally, many phase II and III clinical trials are currently ongoing to assess the safety and efficacy of other potential therapies for the treatment of AML. In this review, we summarize the results of the landmark clinical trials associated with the newly approved agents as well as the current ongoing clinical trials for the treatment of AML. A literature search was performed to retrieve data on agents currently being studied for use. Although the overall prognosis for patients with AML remains poor, the addition of the newly FDA-approved medications is a step in the right direction for a disease state that has proved difficult to treat.

Keywords: AML pipeline; Vyxeos; acute myeloid leukemia; enasidenib; gemtuzumab; midostaurin.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Clinical Trials as Topic
  • Drug Approval
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy*
  • United States
  • United States Food and Drug Administration

Substances

  • Antineoplastic Agents