Revumenib Approved to Treat Certain Cases of Acute Myeloid Leukemias
The FDA has approved the menin inhibitor revumenib for relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation in certain adult and pediatric patients 1 year and older.
The U.S. Food and Drug Administration (FDA) has approved the menin inhibitor revumenib (Revuforj) for patients who have relapsed or refractory acute myeloid leukemia (AML) that harbors a susceptible nucleophosmin 1 (NPM1) mutation, are 1 year of age or older, and who have no satisfactory alternative treatment options.
Revumenib works by binding to a protein called menin to prevent its interaction with the mixed lineage leukemia 1 (MLL1) protein, thereby blocking cancer-promoting gene expression mediated by the menin-MLL1 complex. NPM1 is a protein involved in several critical cellular processes. Mutated forms of NPM1, found in many cases of AML, can enhance oncogenic gene expression by binding to the menin-MLL1 complex, according to a study published in Cancer Discovery, an American Association for Cancer Research (AACR) journal.
Revumenib, the first menin inhibitor to be greenlit by the FDA, was previously approved for treating relapsed or refractory acute leukemias with a different genetic mutation—a KMT2A translocation that produces a mutant form of MLL1.
The latest approval is based on results from the phase II portion of the open-label, multicenter phase I/II AUGMENT-101 trial. The study enrolled 84 patients at least 30 days old with relapsed or refractory NPM1-mutant AML. Enrolled patients were treated with revumenib with or without a strong CYP3A4 inhibitor, which is used to enhance the systemic exposure levels of revumenib.
Complete responses with partial recovery of blood cell counts were observed in 23.1% of efficacy-evaluable patients and lasted for a median of 4.5 months. In addition, eight of the 46 patients who were dependent on blood transfusions at baseline no longer required transfusions for at least 56 days during or after revumenib treatment.
The prescribing information includes a black box warning highlighting the risk of differentiation syndrome, which is the rapid release of cytokines—proteins that affect the immune system—brought on by the treatment-induced maturation of leukemia cells. Another black box warning highlighted in this section is irregular electrical activity in the heart, specifically QTc prolongation and Torsades de Pointes.
If administered with a strong CYP3A4 inhibitor, the recommended dose for revumenib is 160 mg orally twice daily for patients who weigh at least 40 kg or 95 mg/m2 for those who weigh less than 40 kg. If administered without a strong CYP3A4 inhibitor, the dose is 270 mg orally twice a day for patients who weigh 40 kg or more or 160 mg/m2 for those weighing less than 40 kg. Treatment can be continued until disease progression or unacceptable toxicity.
AML is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. According to federal statistics, it was estimated that 22,010 individuals would be diagnosed with AML and 11,090 patients would die of the disease in the United States in 2025.
About 30% of AML patients have NPM1 mutations, which makes it one of the most common subtypes of AML. Approximately 50% of patients with NPM1-mutated AML eventually relapse after receiving the current standard of care, which is primarily chemotherapy based.
The FDA rendered its decision on October 24, 2025. Check this resource for updated information on all therapeutics regulated by the FDA.