FDA Approves New Treatment for R/R Acute Leukemia with KMT2A Translocation: Revumenib

Syndax Pharmaceuticals announced on November 15th, 2024, that a new treatment called revumenib (Revuforj) was FDA-approved for relapsed/refractory acute leukemia patients with the KMT2A translocation. This includes both adult and pediatric patients one year and older. 

Up to 15% of acute leukemia patients have the KMT2A gene translocation in their cancer cells. This affects mostly acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL).  

Learn how revumenib works and how you may be eligible to receive it. 

What is Revumenib? 

Revumenib is the first menin inhibitor in the US for people with KMT2A translocated acute leukemia. It is a pill taken by mouth. 

The KMT2A translocation (lysine methyltransferase 2A gene) means that part of one chromosome breaks and attaches to a different chromosome. This causes an aggressive form of cancer. 

A menin inhibitor works to stop the fusion of KMT2A and menin proteins. Stopping menin and KMT2A from interacting helps change how multiple genes are transcribed, influencing how the cancer cell acts. 

How Effective is Revumenib for Acute Leukemias? 

104 relapsed/refractory acute leukemia patients with a KMT2A translocation were treated with revumenib in the AUGMENT-101 trial. Of these: 

• 21% of patients achieved a full reduction of cancer signs/symptoms (complete remission)
• The average amount of time patients remained in complete remission was 6.4 months
• 23% of patients went on to receive a stem cell transplant after taking revumenib

The study's results help improve the 5% complete remission rate that acute leukemia patients with the KMT2A translocation usually experience. 

Common side effects from revumenib included hemorrhage, nausea, infections, muscle/joint pain, edema, decreased appetite, differentiation syndrome, electrolyte abnormalities, diarrhea, constipation, and fatigue. If you have concerns about how treatment-related side effects are managed, please contact your doctor. 

"The FDA approval of the first menin inhibitor is a major breakthrough for patients with R/R acute leukemia with a KMT2A translocation, a genetic alteration associated with a very poor prognosis. 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." -Ghayas C. Issa, M.D., Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center. 

Ongoing research continues for revumenib’s use in R/R AML with an NPM1 mutation and for newly diagnosed patients. Stay tuned for updated results from the ASH conference with longer follow-up data. 

How Can I Receive Revumenib? 

If you would like to receive revumenib, please contact your acute leukemia specialist. Click here to view AML specialists and here for ALL specialists. 

A support service called SyndAccess is also available to help you during your treatment journey. SyndAccess connects you to a highly experienced and compassionate nurse team who offer one-on-one support every step of the way. This also includes help in answering your questions about navigating insurance while taking revumenib. 

Summary 

The FDA approval of revumenib (Revuforj) introduces a new treatment for acute leukemia patients with the KMT2A gene translocation (such as AML and ALL). 

This first approved menin inhibitor in the US not only extends complete remission periods for a significant portion of patients but also serves as a potential bridge to further curative treatments like stem cell transplants. 

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Sources: 

• Syndax Announces FDA Approval of Revuforj® (revumenib), the First and Only Menin Inhibitor to Treat Adult and Pediatric Patients with Relapsed or Refractory Acute Leukemia with a KMT2A Translocation  
• KMT2A Rearrangements in Leukemias: Molecular Aspects and Therapeutic Perspectives