What Is Revuforj, and Why Does It Matter?
Revuforj (revumenib) is an oral medicine approved for treating a type of cancer called acute myeloid leukemia (AML) that affects your bone marrow and blood. In 2024, Revuforj became the first approved medicine for treating acute leukemia with a different genetic change called a KMT2A translocation. The FDA has now approved Revuforj for adults and children age 1 year or older whose AML has come back or hasn't responded to other treatments and who have a specific change in a gene called nucleophosmin 1 (NPM1).
Revuforj is a first-in-class medicine that works by blocking a protein called menin, which promotes growth of leukemia cells, especially in the presence of certain genetic changes such as KMT2A rearrangements or NPM1 mutations. In NPM1-mutated AML, the faulty NPM1 disrupts normal cell function, causing a fast-growing type of AML that often comes back after treatment. By stopping menin from working with KMT2A protein, Revuforj shuts down abnormal gene signals and allows leukemia cells to grow and mature more normally.
This approval is important because NPM1 mutations are the most common genetic change in AML, found in about 3 out of 10 adults with the condition. The cancer in these cases tends to be more aggressive and harder to treat, especially when it comes back or doesn't respond to treatment. Until now, there were no medicines specifically designed to target this mutation. Special tests are available that can check for NPM1-mutated AML and help patients who may benefit from a more targeted therapy.
Why Was It Approved?
The FDA approved Revuforj because clinical studies showed promising results in treating people with relapsed or hard-to-treat AML with an NPM1 mutation.
In a study, 23% (15 out of 65) patients achieved either a complete remission or a near-complete remission (with some recovery in blood counts), meaning their leukemia improved significantly. On average, it took about 2.8 months to see a response, and the remissions lasted around 4.5 months. Among the 46 patients who needed transfusions at the start of the study, eight of them (17%) no longer needed red blood cell or platelet transfusions during treatment.
The most common side effects of Revuforj include changes in lab test results (such as increased levels of liver enzymes, creatinine, phosphate, triglycerides, alkaline phosphatase, and parathyroid hormone, as well as low potassium and phosphate levels and low white blood cell counts with fever), along with bleeding, viral or bacterial infections, nausea, diarrhea, constipation, fatigue, muscle or joint pain, swelling, and loss of appetite.
Serious side effects including infections, bleeding, differentiation syndrome (a serious reaction in which leukemia cells mature too quickly, causing inflammation and fluid buildup), and dangerous heart rhythm changes were seen in some patients with NPM1-mutated AML. In a few cases, these side effects caused death from sudden heart issues, bleeding, or severe reactions like differentiation syndrome. For these reasons, your doctor will monitor you closely and may adjust or stop treatment if needed.
What Do I Need to Know?
You take Revuforj by mouth twice a day, either without food or with a low-fat meal. Your dose depends on your weight and whether you're taking other medicines that affect how your body processes Revuforj. If you miss a dose or forget to take it on time, take Revuforj as soon as you can, at least 12 hours before your next scheduled dose. Don't take two doses within 12 hours. Go back to your regular dosing schedule the next day.
Swallow the pill whole without cutting or chewing it. If you can't swallow tablets, you can crush them and mix them with water following specific instructions for preparation. Be sure to take the mixture within two hours. If you vomit or don't finish the full dose, don't take another one. Do not stop taking Revuforj or change your dose unless your health care provider tells you to.
Before starting treatment, your white blood cell count needs to be lowered to a safe level. Your health care provider will check your blood counts, electrolyte levels (like potassium and magnesium levels), and heart rhythm regularly, especially during the first month.
Tell your health care provider about all your medical conditions, especially if you have any heart problems such as long QT syndrome and low blood levels of potassium or magnesium. Revuforj should also not be used with other medicines that can affect your heart rhythm. Let your health care provider know about all medications you take, including over-the-counter medicines, vitamins, and herbal supplements, as these medicines may affect Revuforj, causing side effects.
Revuforj can harm an unborn baby. Your health care provider may perform a pregnancy test within seven days before you start treatment. If you're pregnant or could become pregnant, use effective birth control during treatment and for four months after your last dose. Men with partners who could become pregnant should also use birth control during this time and for four months after the last dose. Don't breastfeed while taking Revuforj and for at least a week after your last dose.
Call your health care provider right away if you notice any signs of differentiation syndrome such as fever; dizziness; coughing; trouble breathing; swelling of the arms, legs, neck, groin, or underarm area; a severe headache; confusion; rash; or heart issues such as an irregular or very fast heartbeat during your treatment.
Revuforj is available in the U.S. through specialty pharmacies. If cost is a concern, support and financial assistance may be available for eligible patients.