On October 16, 2020, the U.S. Food and Drug Administration (FDA) granted regular approval to venetoclax (Venclexta) in combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed acute myeloid leukemia (AML) in adults aged 75 or older or who have comorbidities precluding intensive induction chemotherapy. Venetoclax was initially granted accelerated approval for this indication in November 2018. Efficacy was confirmed in two randomized, double-blind, placebo-controlled trials in patients with AML.
Phase III VIALE-A Trial
In VIALE-A, patients were randomly assigned to receive venetoclax plus azacitidine (n = 286) or placebo plus azacitidine (n = 145). Efficacy was established based on an improvement in overall survival. The median overall survival was 14.7
Courtney D. DiNardo, MD, MSCE
months in patients treated with venetoclax plus azacitidine compared to 9.6 months in those receiving placebo plus azacitidine (P < .001). Patients treated with venetoclax plus azacitidine also demonstrated an improvement in complete remission rate: 37% vs 18%.
The phase III VIALE-A trial was reported in The New England Journal of Medicine, by Courtney D. DiNardo, MD, MSCE, of The University of Texas MD Anderson Cancer Center, and colleagues.1
Phase III VIALE-C Trial
Updated data from the VIALE-C trial were presented at the ASCO20 Virtual Scientific Program and reported in Blood, by Andrew H. Wei, MD, of The Alfred Hospital and Monash University, in Melbourne, and colleagues.2,3 In VIALE-C, patients were randomly assigned to receive venetoclax plus low-dose cytarabine or
Andrew H. Wei, MD
placebo plus low-dose cytarabine. As of August 15, 2019, 211 patients had been randomly assigned. At a median follow-up of 17.5 months, the investigators concluded that the venetoclax combination offers a clinically meaningful improvement in overall survival compared to placebo plus low-dose cytarabine and is an effective therapeutic option for first-line treatment of older patients with AML or other patients not able to receive intensive chemotherapy.
1. DiNardo CD, et al: N Engl J Med 383:617-629, 2020.