The U.S. Food and Drug Administration (FDA) has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). The indication is for adult and pediatric patients one year of age and older with a susceptible nucleophosmin 1 (NPM1) mutation who have no effective alternative treatment options.
AML is a rare cancer affecting the blood and bone marrow. The signs and symptoms of AML vary but may include easy bruising, bone pain or tenderness, fatigue, fever, frequent nosebleeds, bleeding from the gums, shortness of breath, and weight loss. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor.
Revumenib is an oral, first-in-class menin inhibitor previously indicated for the treatment of patients relapsed/refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation. The expansion of its indication to AML is based on data from the phase 2 AUGMENT-101 clinical trial, recently published in the Blood journal.
The rate of complete remission (CR) plus complete remission with partial hematological recovery (CRh) was 23%. The median time to CR/CRh response was 2.8 months and the median duration of CR/CRh was 4.5 months. Safety was evaluated in a 241 patient analysis and the most common adverse reactions are consistent with the known safety profile of revumenib.
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