FDA grants full approval of venetoclax for the treatment of newly diagnosed AML

On October 16, 2020, the U.S. Food and Drug Administration (FDA) granted full approval of venetoclax in combination with azacitidine, decitabine, or low dose cytarabine (LDAC) for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. The approval was based on the results of the phase III trials VIALE-A (NCT02993523) and VIALE-C (NCT03069352).¹

The VIALE-A trial showed a 34% reduction in the risk of death in patients treated with the combination of venetoclax + azacitidine compared with placebo + azacitidine (HR, 0.66; 95% CI, 0.52–0.85; p < 0.001). Median overall survival in these two groups was 14.7 and 9.6 months, respectively. Complete remission was also increased in the venetoclax cohort, at a rate of 37% vs 18%.¹ (More detail on the trial results can be found here, including our video interview with Steering Committee member, Courtney DiNardo).

The VIALE-C trial showed an increase in median overall survival with venetoclax in combination with LDAC compared to placebo + LDAC, 7.2 vs 4.1 months (HR, 0.75; 95% CI, 0.52–1.07; p = 0.114), and an increase in complete response rate, 27% vs 7%.¹ (More detail on the trial results can be found here. Alternatively, watch our video interview with Steering Committee member, Andrew Wei, for the 6-month update).

Both combinations were well tolerated with manageable safety profiles. In our video interview below, Daniel Pollyea discusses further the utility of venetoclax in the AML setting, including its use in other subsets of patients.