Originally published by our sister publication Clinical Oncology News
By Clinical Oncology News Staff
The FDA approved revumenib (Revuforj, Syndax), a menin inhibitor, for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year of age and older.
Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700, ClinicalTrials.gov. Identifier: NCT04065399; AUGMENT-101) in 104 adult and pediatric patients (at least 30 days old) with relapsed or refractory acute leukemia with a KMT2A translocation. Patients with an 11q23 partial tandem duplication were excluded. Revumenib was administered until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by four cycles of treatment or hematopoietic stem cell transplantation.
The main efficacy outcome measures were complete remission (CR) plus CR with partial hematologic recovery (CRh), the duration of CR+CRh and conversion from transfusion dependence to independence. The CR+CRh rate was 21.2% (95% CI, 13.8%-30.3%), and the median CR+CRh duration was 6.4 months (95% CI, 2.7 months to not estimable). Of the 22 patients achieving CR or CRh, the median time to CR or CRh was 1.9 months (range, 0.9-5.6 months). Among the 83 patients dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 12 (14%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 21 patients independent of both RBC and platelet transfusions at baseline, 10 (48%) remained transfusion independent 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, ECG QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue and increased alkaline phosphatase.
The full prescribing information for revumenib will be posted here.
Based on a press release from the FDA.
