The FDA has decided not to grant approval to quizartinib for the treatment of patients with FLT3-internal tandem duplication-positive relapsed/refractory (R/R) acute myeloid leukemia, as results of a trial failed to show that the benefits of the treatment outweigh the risks.
At an Oncolytic Drug Advisory Committee (ODAC) meeting in May, panelists concluded in a 3-8 vote, that results of the study failed to show the benefits of quizartinib outweigh the risks for this subgroup of patients.
The panel questioned the lack of robust data, and the large proportion of randomly assigned, untreated patients. The committee also expressed concern over the risk and the modest survival results.
Quizartinib is a small molecule FMS-like tyrosine kinase-3-internal tandem duplication inhibitor that was submitted for approval based on the findings from the phase III QuANTUM-R study. Results from the study showed quizartinib reduced the risk of death by 24% compared with chemotherapy in patients with FLT3-ITD-positive R/R after first-line treatment with or without hematopoietic stem cell transplantation (HSCT).
Prior to the meeting, the FDA conducted its own efficacy analysis and found that the median overall survival (OS) was 26.9 weeks with quizartinib compared with 20.4 weeks with chemotherapy.
In the briefing document, the FDA explained how concerns were raised about the generalizability and credibility of the data.
The FDA is scheduled to make a final decision by the 25th August 2019.