The US Food and Drug Administration (FDA) has approved revumenib for relapsed or refractory acute leukemia with KMT2A translocation in adult and pediatric patients 1 year and older.

The efficacy of revumenib was assessed in a single-arm cohort of the multicenter, open-label AUGMENT-101 trial, involving 104 patients with relapsed or refractory acute leukemia with a KMT2A translocation. Exclusions included those with 11q23 partial tandem duplication. Patients received revumenib until disease progression, unacceptable toxicity, lack of response after four cycles, or hematopoietic stem cell transplantation.

The primary efficacy outcomes included complete remission (CR) plus CR with partial hematologic recovery (CRh), their duration, and transfusion independence. The CR+CRh rate was 21.2% (95% CI, 13.8–30.3), with a median duration of 6.4 months (95% CI, 2.7–NE). The median time to response was 1.9 months. Among 83 transfusion-dependent patients, 14% became transfusion-independent, while 48% of baseline-independent patients maintained their independence.

Adverse reactions reported in ≥20% of patients included nausea, differentiation syndrome, QT prolongation, and hematologic abnormalities, among others. Dosing recommendations vary based on weight and CYP3A4 inhibitor use. Due to delays in the availability of the lowest dose strength for patients under 40 kg, access will be facilitated via an expanded access program.