Pharmacist Medication Insights: Azacitidine for Acute Myeloid Leukemia

Azacitidine (Onureg) was approved by the FDA in September 2020 as a maintenance therapy for acute myeloid leukemia.

A 2020 study found that patients with acute myeloid leukemia (AML) that has gone into remission following initial chemotherapy remained in remission longer and had improved overall survival (OS) if they took a pill form of azacitidine as a maintenance treatment. The randomized, international phase 3 clinical trial showed was the first to show an AML maintenance treatment had such a strong benefit for patients. Azacitidine was quickly adopted in practices as a standard part of care.

Published in The New England Journal of Medicine, the findings led to the FDA approving oral azacitidine (Onureg) in September 2020 as an AML maintenance therapy.

Currently, AML is the most common form of acute leukemia in adults and is a life-threatening hematological cancer. According to the National Cancer Institute, approximately 20,000 people are diagnosed with AML in the United States per year, with 11,000 dying at the same rate.

At the time of diagnosis, patients are usually either middle-aged or older adults. The 5-year OS rate is approximately 30%, with a 10% survival rate for patients older than 65 years of age. There are also frequent relapses among patients with AML, even following complete remission with initial chemotherapy.