The FDA has granted approval to revumenib, marketed as Revuforj, a novel oral medication for treating acute leukemia in patients aged one year and older who have specific genetic mutations. This approval marks a significant advancement in leukemia treatment, offering a new approach that directly targets the underlying genetic causes of the disease.
Mechanism of Action
Revuforj represents a new class of drugs designed to combat leukemia by selectively blocking the menin protein. Menin interacts with the KMT2A gene, a gene frequently associated with aggressive forms of leukemia. By inhibiting this interaction, Revuforj aims to restore normal gene function and impede the proliferation of leukemic cells.
Ghayas C. Issa, MD, associate professor of leukemia at The University of Texas MD Anderson Cancer Center, stated that the approval is a “major breakthrough,” further noting the “significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients”.
Clinical Trial Data
The approval was based on the results of a clinical trial involving 104 patients. The data revealed that 21% of patients achieved complete remission, with the remission lasting approximately six and a half months. The median time to remission was about two months. Furthermore, 24 patients were able to proceed to stem cell transplants following treatment with Revuforj.
Safety and Availability
Common side effects associated with Revuforj include bleeding, infections, immune reactions, swelling, muscle pain, fatigue, appetite loss, nausea, constipation, diarrhea, electrolyte changes, and blood abnormalities, including elevated liver enzymes. Additionally, QT prolongation, a condition affecting the heart's electrical activity, was observed.
Syndax anticipates making 110-milligram and 160-milligram tablets available through specialty distributors and pharmacies this month. Dosages of 25 milligrams for patients under 40 kilograms are expected by early 2025. In the interim, a liquid oral solution will be accessible to patients weighing less than 40 kilograms through an Expanded Access Program.
