FDA advisory committee does not recommend approval for AC 220 in acute myeloid leukemia

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 8 to 3 against approving an NDA for AC 220 (quizartinib), from Daiichi Sankyo, for adult patients with relapsed/refractory FLT3-ITD–positive acute myeloid leukemia (AML). The FDA has conducted its own efficacy analysis, which revealed that the median overall survival was 26.9 weeks (quizartinib) versus 20.4 weeks (conventional chemotherapy). Phase III trial data revealed that the median event-free survival was 1.4 months (quizartinib) versus 0.9 months (salvage chemotherapy).

In addition, an internal FDA analysis failed to demonstrate a significant event-free survival benefit with quizartinib compared to chemotherapy (6.0 vs. 3.7 weeks respectively). The trial data showed that the overall response rate was 69% (quizartinib) versus 30% (salvage chemotherapy). The composite complete remission rate was 48% versus 27% and the partial response rate was 21% versus 3%, respectively. The median duration of composite complete remission was 12.1 weeks versus 5.0 weeks, respectively. The FDA review pointed to concerns over the credibility and generalizability of the trial data. Now the FDA will make a final decision by 25 August 2019.