FDA approves Revumenib-Revuforj for the treatment of relapsed or refractory acute myeloid leukemia with susceptible NPM1 mutations

On October 24, 2025, the U.S. Food and Drug Administration (FDA) approved Syndax Pharmaceuticals' Revuforj (Revumenib)-Revuforj for the treatment of relapsed or refractory acute myeloid leukemia (AML) patients with susceptible NPM1 mutations. This approval applies to adult and pediatric patients 1 year of age and older who have no effective alternative treatment options.

1. Revumenib’s approval background and innovation

Revumenib is a menin inhibitor. As a new targeted therapy, it exerts a specific effect on NPM1 mutations in AML by inhibiting the menin-MLL complex. The approval of this drug is an important breakthrough in the field of cancer treatment, providing a new treatment option for acute myeloid leukemia (especially patients with NPM1 mutation-positive patients) and filling the treatment gap.

2. Efficacy and safety evaluation

The efficacy and safety of revumenib were evaluated in an open-label, multicenter clinical trial (SNDX-5613-0700, NCT04065399; AUGMENT-101). In this study, patients received Revumenib and their susceptibility to NPM1 mutations was verified by next-generation sequencing (NGS) or polymerase chain reaction (PCR).

Main efficacy indicators:

1. Complete remission rate (CR): 23.1% of patients achieved complete remission, which means that leukemia cells have almost completely disappeared.

2. Complete remission with partial hematological recovery (CRh): This proportion is 23.1% (95%CI: 13.5, 35.2), indicating the effectiveness of treatment.

3. Duration of response:The median duration of CR+CRh is 4.5 months (95% CI: 1.2, 8.1), which provides clinicians with information on the durability of patient efficacy.

4. Improvement in transfusion dependence:Of the 46 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 17% (8 patients) became transfusion independent within 56 days after treatment.

3. Precise dosage and medication specifications

Drug dosage is stratified by weight+CYP3A4 inhibitor synergistic adjustment plan:

1) Weight <40kg and not using strong CYP3A4 inhibitors: 160mg/m² twice daily

2) Weight <40kg, use strong CYP3A4 inhibitor: 95mg/m² twice daily

3) Weight≥40kg without strong CYP3A4 inhibitor: 270mg twice daily

4) Weight≥40kg use strong CYP3A4 inhibitor: 160mg twice daily

Treatment Duration and Monitoring

The use of Revumenib needs to be determined based on the patient's clinical response. Patients should continue to use this drug under the guidance of a physician until disease progression or unacceptable toxicity occurs. If the above situation does not occur, treatment should be carried out for at least 6 months to observe the effect.

4. Safety warning and risk control

The safety of Revumenib has received widespread attention. The FDA warns that it may cause the following adverse reactions:

1. Differentiation syndrome: This complication is relatively common in leukemia treatment and may be life-threatening in severe cases. Patients should seek medical treatment promptly when they experience relevant symptoms.

2. QTc interval prolongation and torsade de pointes syndrome: These heart-related side effects require special attention, and regular electrocardiogram monitoring is required during use.

3. Embryo-Fetotoxicity: Pregnant and lactating female patients should avoid using this drug because it may have adverse effects on the fetus or infant.

As the first menin inhibitor approved by the FDA, revumenib not only fills the gap in the treatment of NPM1 mutant AML, but also creates a new paradigm of "mutation-specific targeted therapy". The approval of its pediatric indication is particularly landmark and addresses the long-standing lack of treatment options in pediatric hematological malignancies. With the accumulation of real-world data, this drug is expected to further optimize treatment strategies and bring survival hope to more patients with relapsed/refractory AML.

Reference: updated onOctober 24, 2025, https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-myeloid-leukemia-susceptible-npm1-mutation