Officials with the FDA recently approved the menin inhibitor revumenib (Revuforj; Syndax Pharmaceuticals, Inc.) for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation.1
The approval, which is for adult and pediatric patients who are at least 1 year old, followed an evaluation of the treatment in AUGMENT-101, an open-label, multicenter trial (NCT04065399) conducted with 104 adult and pediatric patients with relapsed or refractory acute leukemia and a KMT2A translocation. The researchers did not include patients with an 11q23 partial tandem duplication. Revumenib was administered until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by 4 cycles of treatment, or hematopoietic stem cell transplantation.
The researchers noted that the complete remission (CR) and CR with partial hematologic recovery (CRh) rate was 21.2% (95% confidence interval [CI], 13.8-30.3), and the median CR+CRh duration was 6.4 months (95% CI: 2.7, not estimable). Of 22 patients who achieved CR or CRh, the median time to CR or CRh was 1.9 months.
Among 83 patients dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 12 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 remained transfusion-independent during any 56-day post-baseline period.
“The significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients," Ghayas C. Issa, MD, associate professor of leukemia at The University of Texas MD Anderson Cancer Center said in a press release about the approval.2
The most commonly reported adverse reactions, according to an FDA statement, “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.”
References
- FDA. FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation. Published November 15, 2024. Accessed November 19, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda- approves-revumenib-relapsed-or-refractory-acute-leukemia-kmt2a-translocation
- Syndax Pharmaceuticals, Inc. Syndax announces FDA approval of Revuforj (revumenib), the first and only menin inhibitor to treat adult and pediatric patients with relapsed or refractory acute leukemia with a KMT2A translocation. Press release. Published November 15. 2024. Accessed November 19, 2024. https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-first-and-only