Breakthrough therapy designation granted to revumenib by the FDA

On December 5, 2022, revumenib, a potent, selective, small molecule inhibitor of the menin-mixed lineage leukemia (MLL) binding interaction, was granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with relapsed/refractory acute leukemia, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), with KMT2A rearrangements.¹

This designation is based on data from the phase I part of the AUGMENT-101 trial (NCT04065399). The study design and initial data and additional results from the AUGMENT-101 trial have previously been covered by the AML Hub. Briefly, patients with ALL or AML and KMT2A rearrangements and patients with NPM1-mutated AML were included in the trial. At the March 2022 data cutoff, the rate of complete remission or complete remission with partial hematological recovery was 27% for patients with KMT2A rearranged-acute leukemia who received the recommended phase II dose. No patients discontinued treatment due to treatment-related adverse events.¹

Revumenib continues to be investigated in several trials, including the phase II part of the AUGMENT-101 trial.