Attacking FLT3 Mutations Yields First Targeted Therapy in AML

Anita T. Shaffer and Jason M. Broderick
Published: Thursday, Jun 15, 2017
Elias Jabbour, MD

Elias Jabbour, MD

Although FLT3 mutations are well established as a prognostic marker in patients with acute myeloid leukemia (AML), efforts to target the aberration therapeutically were underway for more than 15 years before yielding success. That happened in April, when the FDA approved midostaurin (Rydapt), the first new therapy for AML in about 40 years.


“After a dearth of new therapies available for acute myeloid leukemia over the last few decades, we are transitioning into a new era with several promising strategies in latestage development.”

Table. Selected Later-Stage Clinical Trials of FLT3 Inhibitors in AML


Pivotal Midostaurin Findings

The midostaurin approval is based on the phase III RATIFY trial in AML and 2 single-arm, open-label studies of patients with SM. In RATIFY, the addition of midostaurin to standard chemotherapy reduced the risk of death by 23% compared with chemotherapy alone in patients with FLT3-mutant AML. After censoring for patients who received stem cell transplants, the overall survival (OS) benefit with midostaurin remained steady at 25%.
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