Syndax's Revuforj Gains FDA Approval for KMT2A Leukemia Amid Earlier Setback

Syndax Pharmaceuticals has secured FDA approval for Revuforj, marking a significant advance in the treatment of relapsed or refractory acute leukemia bearing KMT2A gene translocations in patients aged one and older[1][2]. Revuforj is notable for being the first menin inhibitor approved in the U.S., with the label acknowledging a notable 21% complete remission rate in clinical trials[1][2]. The FDA's decision arrived ahead of schedule, underscoring the urgency and promise associated with this new treatment option, despite the inclusion of a black box warning for differentiation syndrome[2].