FDA Approves Revumenib for Adults With KMT2A-Translocated Acute Leukemia

The US Food and Drug Administration on Tuesday approved revumenib, marketed as Revuforj by Syndax Pharmaceuticals, for the treatment of adults with relapsed or refractory acute leukemia bearing a KMT2A translocation. The approval covers both acute myeloid leukemia and acute lymphoblastic leukemia, and is the first menin inhibitor approved for a population that has historically had a five-year survival rate below 25%.

The Clinical Data

The approval is based on the AUGMENT-101 trial, which enrolled 196 adult patients across 38 sites in the United States, Canada, and Europe. The overall response rate was 64%, with a complete remission rate of 38% and a median duration of response of 6.4 months. The data were published in the New England Journal of Medicine in May and reviewed under the FDA's Real-Time Oncology Review program, which cut the standard review timeline roughly in half.

「This is the first targeted therapy for KMT2A-translocated leukemias. The community has been waiting for this for thirty years,」 said Dr. Ghayas Issa of the MD Anderson Cancer Center, the trial's lead investigator.

Why the Translocation Matters

The KMT2A translocation is present in roughly 5% of adult AML cases and 8% of adult ALL cases, with a much higher prevalence — up to 80% — in infant leukemia. Patients with the translocation have historically responded poorly to standard chemotherapy and have not been eligible for many of the targeted therapies that have transformed the treatment of other leukemia subtypes. Revumenib works by inhibiting the menin protein, which the KMT2A fusion requires to drive leukemic transformation.

What's Next

Syndax expects the drug to be available in the United States by mid-August. Pricing has not yet been disclosed, but the company has indicated it will be in line with other recent menin inhibitors and FLT3 inhibitors. Trials in pediatric and front-line settings are ongoing.