Dr. DiNardo on the FDA Approval of Oral Azacitidine in AML

Courtney DiNardo, MD, MSCE, clinical researcher in the Department of Leukemia of the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses the FDA approval of oral azacitidine (CC-486) in acute myeloid leukemia (AML).

On September 1, 2020, the FDA approved oral azacitidine for the continued treatment of adult patients with AML who achieved first complete remission (CR) or CR with incomplete blood count recovery following intensive induction chemotherapy who are not able to complete intensive curative therapy.

The regulatory decision is based on data from the pivotal phase 3 QUAZAR AML-001 trial in which CC-486, an oral formulation of azacitidine, lengthened median overall survival (OS) by 9.9 months versus placebo in patients with AML who were in first remission.

At a median follow-up of 41.2 months, the median OS was 24.7 months (95% CI, 18.7-30.5) with CC-486 versus 14.8 months (95% CI, 11.7-17.6) with placebo; this translated to a 31% reduction in the risk of death with the hypomethylating agent (HR, 0.69; 95% CI, 0.55-0.86; P = .0009). Moreover, the median relapse-free survival (RFS) was 10.3 months versus 4.8 months with CC-486 versus placebo, respectively (HR, 0.65; 95% CI, 0.52-0.81; P = .0001).

The approval is profoundly impactful for patients, says DiNardo. Having an oral and well-tolerated agent that improves survival is the next step the field has been waiting for, concludes DiNardo.