The U.S. FDA granted breakthrough therapy designation to midostaurin, an oral, multi-targeted kinase inhibitor for adults with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) who are eligible to receive standard induction and consolidation chemotherapy.
The agency's decision was based on the results of the phase III RATIFY clinical trial, the results of which were presented during a plenary session at the 2015 ASH Annual Meeting. The study found that patients treated with midostaurin and standard induction and consolidation chemotherapy had a significant improvement in overall survival (OS), compared with those who received standard induction and consolidation chemotherapy alone (hazard ratio = 0.77; p=0.0074). The median OS for the midostaurin treatment cohort was 74.7 months (95% CI 31.7-not reached) compared with 25.6 months for the standard treatment group (95% CI 18.6-42.9).
No statistically significant differences were observed between the treatment groups in terms of grade 3 or higher adverse events. Thirty-seven patient deaths were recorded, though no differences were observed between patient cohorts.
Source: Novartis press release, February 19, 2016.