Rydapt (midostaurin) Phase III RATIFY (CALGB 10603 [Alliance]) data in acute myeloid leukemia published in The New England Journal of Medicine- Novartis

Novartis announced that full results from the Rydapt (midostaurin) Phase III RATIFY (CALGB 10603 [Alliance]) clinical trial in acute myeloid leukemia were published in The New England Journal of Medicine (NEJM). In RATIFY, patients aged 18-59 years treated with Rydapt in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy experienced significant improvement in overall survival (OS) with a 22% reduction in the risk of death compared with chemotherapy plus placebo. In patients in the Rydapt arm, OS was 74.7 months vs. 25.6 months in the placebo arm. At four years, OS was 51.4% in the Rydapt arm, compared with 44.3% in the placebo arm.

The median event-free survival (EFS) was 8.2 months in the Rydapt arm and 3.0 months in the placebo arm. Median DFS was greater with the addition of Rydapt versus the placebo arm (26.7 months vs. 15.5 months, respectively). The complete remission (CR) rate, defined as CR reported within 60 days of protocol therapy initiation, was 58.9% in the Rydapt arm and 53.5% in the placebo arm. The benefit of Rydapt on OS and EFS was consistent across all FMS-like tyrosine kinase 3 (FLT3) mutation subgroups, including internal tandem duplication (ITD) and tyrosine kinase domain (TKD) FLT3 mutations.

Top-line data from this study were previously presented during the plenary session at the American Society of Hematology (ASH) Annual Meeting in 2015. New data include disease-free survival (DFS), further analysis of patients undergoing transplant and expanded safety information.

See: "Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation." Stone RM, Mandrekar SJ, Sanford BL, et al. N Engl J Med. 2017 June 23. doi: 10.1056/NEJMoa1614359. [Epub ahead of print].