What Is Revumenib?

Revumenib (sold under the brand name Revuforj) is a menin inhibitor used to treat certain types of acute leukemia. It was developed by Syndax Pharmaceuticals and received FDA approval in November 2024.

Revumenib is specifically approved for:

• Treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients 1 year and older
• Treatment of relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation in patients who have no satisfactory alternative treatment options (expanded indication, October 2025)

This medication targets a very specific subset of leukemia patients whose cancer is driven by certain genetic mutations, making it an important precision medicine option for patients with limited treatment alternatives.

How does Revumenib work?

Revumenib works by blocking the interaction between two proteins: menin and KMT2A (MLL) fusion proteins. In certain types of leukemia, the KMT2A gene becomes fused with other genes through chromosomal translocations, creating abnormal fusion proteins. These fusion proteins bind to menin and together drive the uncontrolled growth of leukemia cells.

By inhibiting this protein-protein interaction, Revumenib disrupts the transcription of genes that promote leukemia cell growth and survival. This leads to antiproliferative and antitumor activity, effectively slowing or stopping the growth of leukemia cells that harbor KMT2A fusions or NPM1 mutations.

What doses are available for Revumenib?

• 25 mg tablets — primarily for pediatric dosing, available since early 2025
• 110 mg tablets — available since FDA approval in November 2024
• 160 mg tablets — available since FDA approval in November 2024

All formulations come in bottles of 30 tablets. The typical adult dose is 270 mg twice daily, which may be reduced to 160 mg twice daily when taken with strong CYP3A4 inhibitors.

How hard is it to find Revumenib in stock?

Revumenib (brand name Revuforj) scores a 35 out of 100 on our findability scale, meaning it is quite difficult to locate. As a specialty oncology medication, Revumenib is only available through specialty pharmacies — you will not find it at your local retail pharmacy like CVS or Walgreens. Distribution is tightly controlled through Syndax Pharmaceuticals' specialty pharmacy network. While there is no formal FDA-listed shortage, the restricted distribution model means patients must go through specific channels to obtain this medication.

Who Can Prescribe Revumenib?

Revumenib is prescribed exclusively by specialists with expertise in blood cancers. The types of doctors who prescribe this medication include:

• Hematologists — doctors who specialize in blood disorders
• Oncologists — cancer specialists
• Hematology-Oncologists — specialists in both blood disorders and cancer
• Pediatric Oncologists — for eligible patients aged 1 year and older

This is not a medication that a primary care physician or general practitioner would prescribe. Patients must have confirmed genetic testing showing a KMT2A translocation or NPM1 mutation before treatment can begin. Revumenib is not available through telehealth services.

Is Revumenib a controlled substance?

No, Revumenib is not a controlled substance and does not have a DEA schedule. However, it does carry two FDA boxed warnings — the most serious type of safety warning:

• Differentiation syndrome — a potentially fatal condition that requires immediate medical treatment
• QTc prolongation and Torsades de Pointes — life-threatening cardiac arrhythmias that require regular ECG monitoring

Because of these serious risks, patients require close medical supervision including ECG monitoring before starting treatment, weekly for the first 4 weeks, and monthly thereafter.

Common Side Effects of Revumenib

Revumenib can cause a range of side effects. The most commonly reported include:

• Hemorrhage/bleeding
• Nausea
• Musculoskeletal pain
• Infection
• Febrile neutropenia (fever with low white blood cell count)
• Diarrhea
• Decreased appetite
• Constipation
• Edema (swelling)
• Fatigue

Serious side effects that require immediate medical attention include differentiation syndrome (which can be fatal), QTc prolongation, severe hemorrhage, and severe infections. Contact your healthcare provider immediately if you experience fever, difficulty breathing, rapid weight gain, chest pain, or irregular heartbeat.

Alternative Medications to Revumenib

Revumenib is one of a new class of menin inhibitors. Alternative medications in this class include:

• Ziftomenib — another menin inhibitor in late-stage clinical trials (KOMET-001) for KMT2A-rearranged and NPM1-mutated AML, showing meaningful efficacy at the 600 mg dose in heavily pretreated patients
• Bleximenib (JNJ-75276617) — a menin inhibitor developed by Johnson & Johnson currently in clinical evaluation for acute leukemia with KMT2A rearrangements
• Enzomenib (DSP-5336) — a menin inhibitor in clinical trials being evaluated as monotherapy and in combination regimens for acute leukemia

Note that these alternatives are currently in clinical trials and may not yet be commercially available. Your oncologist can help determine if a clinical trial may be appropriate for your situation.

Drug Interactions with Revumenib

Revumenib has several important drug interactions to be aware of:

• Strong CYP3A4 inhibitors (Posaconazole, Itraconazole, Voriconazole, Cobicistat) — these increase Revumenib exposure and require a dose reduction to 160 mg twice daily
• Strong or moderate CYP3A4 inducers — these may decrease Revumenib levels, reducing its effectiveness or increasing the risk of QT prolongation from its metabolite; avoid concomitant use
• QTc-prolonging drugs — avoid taking with other medications that can prolong the QT interval; if unavoidable, ECGs must be monitored closely

Food interaction: Revumenib must be taken either fasting or with a low-fat meal only (no more than 400 calories and no more than 25% fat). High-fat meals can affect how the drug is absorbed.

Final Thoughts on Revumenib

Revumenib (Revuforj) represents a significant advance in treating relapsed or refractory acute leukemia driven by KMT2A translocations and NPM1 mutations. As a first-in-class menin inhibitor, it offers hope for patients with very limited treatment options.

While the medication is expensive at approximately $39,500 per month, the Revuforj Copay Program through SyndAccess can reduce out-of-pocket costs to as little as $0 for eligible commercially insured patients. For patients needing additional support, SyndAccess also provides a dedicated nurse navigator and financial assistance resources. Contact SyndAccess at 1-888-567-SYND (7963) for more information.

Because Revumenib is only available through specialty pharmacies, patients should work closely with their oncology team and SyndAccess to ensure timely access to this medication.