FDA Approves First Menin Inhibitor for Relapsed, Refractory Acute Leukemia With KMT2A Translocation

Revumenib (Revuforj^®) was approved by the US Food and Drug Administration (FDA) as the first menin inhibitor to treat adult and pediatric patients with relapsed or refractory acute leukemia with a KMT2A translocation, according to a press release from Syndax, the manufacturer of the drug. Revumenib will be available for order in the United States beginning November 2024.

The approval is based on results of the phase I/II AUGMENT-101 trial, where revumenib achieved a complete remission (CR) plus CR with partial hematologic recovery (CRh) rate (CR+CRh) of 21% (95% CI, 13.8-30.3%); a median duration of CR+CRh of 6.4 months (95% CI, 2.7-not estimable); and a median time to CR or CRh of 1.9 months (0.9-5.6 months). Twenty-three percent of patients proceeded to hematopoietic stem cell transplantation following treatment.

Due to adverse events (AEs), 10% of patients had dose reductions and 12% discontinued treatment. Common AEs included hemorrhage, nausea, musculoskeletal pain, infection, febrile neutropenia, diarrhea, and differentiation syndrome.

“FDA approval of the first menin inhibitor is a major breakthrough for patients with [relapsed or refractory] acute leukemia with a KMT2A translocation, a genetic alteration associated with a very poor prognosis,” Ghayas C. Issa, MD, an Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center and study investigator, said in the press release.

He concluded, “The significant clinical benefit and robust efficacy seen with [revumenib] 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.”

Read more: AUGMENT-101: Revumenib Shows ‘Clinically Meaningful Results’ in KMT2Ar AML, ALL