What is the name of the drug and what does it do?
Revuforj (pronounced REV-you-forge), generic name revumenib, is a prescription medication used to treat adults and children aged 1 year and older with relapsed or refractory acute leukemia. This treatment is specifically for cases with a lysine methyltransferase 2A (KMT2A) gene translocation, a genetic abnormality linked to certain aggressive leukemias. Revuforj was approved on November 15, 2024.
How does it work?
Revuforj is a menin inhibitor, which works by blocking the interaction between the KMT2A fusion protein and menin. This interaction is a key driver of cancer cell growth in KMT2A-translocated leukemia. By disrupting this process, Revuforj helps stop the progression of leukemia and encourages the cancer cells to die or differentiate into normal cells.
What did the research discover?
In a clinical study with 104 patients, Revuforj demonstrated significant effectiveness in treating relapsed or refractory acute leukemia with KMT2A translocation.
- 21% of patients achieved complete remission (CR) or complete remission with partial hematologic recovery (CRh).
- Among these, the median time to achieve remission was 1.9 months, and the median duration of remission was 6.46 months.
- Additionally, some patients dependent on blood transfusions at the start of the trial became independent of transfusions during treatment.
These results show Revuforj’s potential to improve outcomes in a challenging group of leukemia patients.
What are some of the side effects?
The most common side effects of Revuforj include:
- Bleeding (hemorrhage)
- Nausea and vomiting
- Muscle pain
- Fatigue
- Low appetite
- Swelling in arms and legs
- Infections, including bacterial and viral infections
- Diarrhea and constipation
What are the dosage recommendations and how is it prescribed?
Revuforj is taken as oral tablets twice daily, approximately 12 hours apart, either without food or with a low-fat meal.
- For patients weighing 40 kg or more, the recommended dose is 270 mg twice daily.
- For patients under 40 kg, dosing is based on body surface area (BSA).
Patients should follow their doctor’s instructions and avoid cutting or chewing the tablets. If needed, the tablets can be crushed and dissolved in water for easier administration.
Source
U.S. Food and Drug Administration. REVUFORJ (revumenib) Tablets: Highlights of Prescribing Information [Internet]. 2024 [cited 2025 Oct 06]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218944s000lbl.pdf
