What medicine is Revuforj (Revumenib)?

Revuforj (Revumenib) is a first-in-class menin inhibitor indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year of age and older. Rearrangements of the KMT2A gene (KMT2Ar) lead to an aggressive form of acute leukemia that is associated with a very poor prognosis and high relapse rates. KMT2Ar gene abnormalities are found in approximately 10% of acute leukemias.

Revumenib, the main ingredient in Revuforj, treats KMT2A acute leukemia by blocking the interaction of wild-type lysine methyltransferase 2A (KMT2A) and KMT2A fusion proteins with menin, a scaffold protein that controls gene expression and cell signaling. The U.S. Food and Drug Administration (FDA)approval ofRevuforj is based on positive data from the AUGMENT-101 clinical trial. The rate of complete remission plus complete remission with partial hematological recovery (CR+CRh) was 21.2%, the median duration of CR+CRh was 6.4 months, and the median duration of CR or CRh was 1.9 months.

Revuforj tablets are administered orally twice daily without food or with a low-fat meal. Common adverse reactions (≥20%) include bleeding, nausea, musculoskeletal pain, infection, febrile neutropenia, bacterial infection, diarrhea, differentiation syndrome, QTc interval prolongation, decreased appetite, constipation, edema, viral infection, and fatigue. Revuforj warns about differentiation syndrome, which can be fatal. Warnings and precautions related to Revuforj include QTc interval prolongation and embryo-fetal toxicity.

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