The FDA recently approved Revuforj (revumenib) for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. Revuforj is the first menin inhibitor to be approved.
In clinical trials:
- About 21% of patients had their cancer go into remission
- For those who responded, the remission lasted about 6.4 months on average
- Some patients were able to stop needing blood transfusions
Side effects
The most common side effects included:
- Bleeding
- Nausea
- Changes in blood tests (like phosphate and liver enzyme levels)
- Muscle and bone pain
- Infections
- Fever with low white blood cell counts
What’s next
- Revuforj will be available soon for most patients
- For very small children (under 40 kg), it may be available through a special program at first
This approval gives patients with this specific type of leukemia a new, targeted treatment option when other therapies have not worked. It is an exciting step forward in personalized cancer care.
About Revuforj: Targeting the Menin-KMT2A Interaction
Revuforj is a potent and selective oral inhibitor that disrupts the interaction between two key proteins: menin and KMT2A (also known as MLL1). This protein complex plays a crucial role in leukemogenesis, particularly in acute leukemias with KMT2A rearrangements or NPM1 mutations.
Blocking Protein Binding: Revuforj acts as a wedge, preventing menin from binding to KMT2A. This disruption is critical because the menin-KMT2A complex is often described as the “key that starts the engine of leukemia cell production.” The disruption of the menin-KMT2A interaction by Revuforj results in two primary outcomes for leukemia cells:
· Cell Differentiation: Leukemia cells that were previously arrested in an immature state begin to differentiate into normal cells
· Cell Death: Some leukemia cells undergo apoptosis (programmed cell death) when deprived of the menin-KMT2A interaction.
More Reading
Join the conversation on CancerConnect: Leukemia Discussions
References:
Issa GC, et al. Menin Inhibition with Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101). J Clin Oncol. Published online August 9, 2024.
Nguyen D, et al. Early mortality in acute myeloid leukemia with KMT2A rearrangement is associated with high risk of bleeding and disseminated intravascular coagulation. Cancer. 2023;129(12):1856-1865.
