Revumenib receives FDA approval for adult and pediatric patients aged ≥1 year with R/R acute leukemia and a KMT2A translocation

On November 15, 2024, the U.S. Food and Drug Administration (FDA) approved revumenib, a menin inhibitor, for the treatment of adult and pediatric patients aged ≥1 year with relapsed/refractory (R/R) acute leukemia with a KMT2A translocation. This approval is based on results from the AUGMENT-101 trial (NCT04065399).¹

AUGMENT-101 trial

The AUGMENT-101 trial is a multicenter, single arm, open-label, phase I/II study investigating the safety and efficacy of revumenib in patients with acute leukemia, including those with an ​MLL/KMT2A gene rearrangement or NPM1 mutation.² Among the evaluable adult and pediatric patients (≥30 days old) with R/R acute leukemia and a KMT2A translocation (n = 104), the key outcomes were¹:

• The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.2% (95% confidence interval [CI], 13.8–30.3), with a median duration of 6.4 months (95% CI, 2.7, not estimable).
• Among the 22 patients who achieved CR or CRh, the median time to reach CR/CRh was 1.9 months (range, 0.9–5.6 months).
• Out of 83 patients who required red blood cell (RBC) and/or platelet transfusions at the baseline, 12 became transfusion free during any 56-day post-baseline period.
• Of the 21 patients independent of both RBC and platelet transfusions at baseline, 10 remained transfusion free during any 56-day post-baseline period.

The most common adverse reactions (≥20%) were hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.