FDA Approval: Revumenib (Revuforj) for Acute Leukemia

A first-of-its-kind target

Some acute leukemias are driven by a rearranged gene called KMT2A. A translocation means a piece of one chromosome has broken off and joined another. The result is an abnormal gene. Leukemia with this change can come back after treatment, which is called relapsed. It can also fail to respond at all, which is called refractory. Both situations have been very hard to treat.

Revumenib, sold as Revuforj, is the first approved drug in a new class called menin inhibitors. It is a tablet that works by blocking a protein called menin. On November 15, 2024, the FDA approved it under new drug application 218944.

Exactly who the approval covers

The FDA wording is specific: revumenib is approved for "relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older."

Read that narrowly. The leukemia must carry the KMT2A translocation, found by testing the leukemia cells. The disease must be relapsed or refractory, so this is not a first treatment. The approval also reaches children as young as 1 year. That matters because this form of leukemia occurs in children as well as adults.

The approval covers acute leukemias with this translocation. In the trial, that included acute myeloid leukemia, acute lymphoblastic leukemia, and mixed-phenotype forms. It does not cover leukemias without the KMT2A change.

What the trial showed, and what it did not

The approval rested on AUGMENT-101, a single-arm study. Everyone received revumenib; there was no comparison group.

The study included 104 adult and pediatric patients with this leukemia. Of them, 21.2 percent reached complete remission, or complete remission with partial blood count recovery. Together these are called CR+CRh. The 95 percent confidence interval was 13.8 to 30.3 percent. The median time to that response was 1.9 months. The median duration of response was 6.4 months. Of 83 patients who needed transfusions at the start, 12, or 14 percent, became transfusion independent for a period during treatment.

Two limits matter. First, a remission rate is not proof of living longer. Without a comparison group, the study cannot show how these patients would have done on other care. Second, most patients in the trial did not reach CR or CRh. For a heavily pretreated leukemia, that response was still enough for FDA approval. It is a measured result, not a cure headline.

A boxed warning and real side effects

The label carries a boxed warning, FDA's most prominent type, for differentiation syndrome. This reaction can be fatal. The label tells doctors to start corticosteroid treatment right away if it is suspected. In the trial's safety group, it occurred in 39 of 135 patients, or 29 percent. It was severe in 13 percent, and one case was fatal.

QTc prolongation is a change in the heart's electrical rhythm seen on an ECG. It also occurred in 29 percent of patients. In 8 percent, the interval passed 500 milliseconds. Heart-rhythm checks, and care with other drugs that affect QT, are part of treatment.

The label's most common side effects include bleeding (53 percent), nausea (51 percent), and raised phosphate (50 percent). Muscle and bone pain affected 42 percent, and infection 41 percent. Liver enzyme rises showed up in 37 and 33 percent for AST and ALT. Febrile neutropenia, a fever with low infection-fighting cells, affected 35 percent.

The recommended dose depends on body weight and other medicines. Patients weighing 40 kilograms or more take 270 milligrams twice daily. That drops to 160 milligrams twice daily with strong CYP3A4 inhibitors, drugs that slow revumenib's breakdown. Doses for smaller patients are set by body size. Follow your own prescription, not this page.

The 2025 expansion to NPM1-mutated AML

On October 24, 2025, FDA approved revumenib for a second group: "relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options."

That approval also came from AUGMENT-101. In the NPM1 group, the CR+CRh rate was 23.1 percent, with a 95 percent confidence interval of 13.5 to 35.2. The median duration was 4.5 months. Note the extra condition in the wording. This use is for people with no satisfactory alternative treatment options.

What to raise with the team

• Has my leukemia been tested for a KMT2A translocation or an NPM1 mutation, and what did the report say?
• Given my prior treatments, do I fit inside either approval's wording?
• What monitoring will I need for differentiation syndrome and heart rhythm, and which symptoms should trigger a same-day call?
• How would this option compare with a clinical trial or a transplant plan in my case?
• Which of my current medicines could interact with revumenib?

What this does not mean

• This is not a treatment for leukemia in general. It applies only to acute leukemia with a KMT2A translocation, or, under the 2025 approval, AML with a susceptible NPM1 mutation.
• It is not a first treatment. Both approvals cover relapsed or refractory disease.
• A 21.2 percent remission rate means most trial patients did not reach complete remission. Response is not a guarantee.
• The trial had no comparison group. It does not show that revumenib helps people live longer than other options.
• Approval wording describes a group. Whether the drug fits one person depends on testing, prior treatment, and that person's own goals.

Sources

• FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation
• FDA prescribing information: REVUFORJ (revumenib) tablets, NDA 218944
• FDA approves revumenib for relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation