FDA approves revumenib for certain patients with advanced acute leukemia
The FDA approved revumenib for the treatment of certain patients with advanced acute leukemia.
The indication applies to use of the agent by adults or by children aged at least 1 year who have relapsed or refractory disease and a lysine methyltransferase 2A gene (KMT2A) translocation, a genetic alteration associated with poor prognosis.
Revumenib (Revuforj, Syndax Pharmaceuticals) is an oral menin inhibitor.
The FDA based approval on data from the phase 1/phase 2 AUGMENT-101 trial, which included 104 adults and children with relapsed or refractory acute leukemia with a KMT2A translocation. Researchers excluded patients with an 11q23 partial tandem duplication.
Patients received revumenib until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by four cycles of treatment or receipt of hematopoietic stem cell transplantation, according to an FDA press release.
Complete remission (CR), complete remission with partial hematologic recovery (CRh), duration of CR and CRh, and conversion from transfusion dependence to independence served as the main efficacy outcomes.
Researchers reported a CR+CRh rate of 21.2% (95% CI, 13.8-30.3), and a median CR+CRh duration of 6.4 months (95% CI, 2.7 to not estimable).
Twenty-two patients achieved CR or CRh. Median time to CR or CRh was 1.9 months (range = 0.9-5.6).
For the 83 patients dependent on transfusions at baseline, 12 (14%) became independent during any 56-day post-baseline period. Among the 21 patients independent of transfusions at baseline, 10 (48%) 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 Syndax Pharmaceuticals press release.
Adverse reactions that occurred among at least 20% of study participants included hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, QT prolongation, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue and increased alkaline phosphatase.
The FDA previously granted priority review, breakthrough designation and orphan drug designation to revumenib or this indication.
References:
- FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation (press release). Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-leukemia-kmt2a-translocation. Published Nov. 15, 2024. Accessed Nov. 18, 2024.
- 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). Available at: https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-first-and-only. Published Nov. 15, 2024. Accessed Nov. 18, 2024.
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