Delivery 7–10 daysRevuforj (revumenib) 110 mg tablets
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Description
💊 Prescription menin inhibitor for relapsed or refractory acute leukaemia with a KMT2A translocation in patients from one year of age, and for relapsed or refractory acute myeloid leukaemia with a susceptible NPM1 mutation where no satisfactory alternative exists.
▪︎ Revumenib 110 mg film-coated tablets, bottle of 30; taken every 12 hours in 28-day cycles (270 mg twice daily from 40 kg, or 160 mg twice daily with a strong CYP3A4 inhibitor)
▪︎ Manufacturer: Syndax Pharmaceuticals
▪︎ FDA approval: November 2024, indication extended in 2025
🔬 How it works: in KMT2A-rearranged leukaemia the fusion protein keeps the genes of cellular immaturity switched on, so the cell is stuck as a blast and cannot mature. It reaches that DNA with the help of menin, an ordinary cellular protein acting as its guide. Revumenib wedges between menin and KMT2A and breaks the bond: the immaturity programme switches off and leukaemic cells begin maturing into normal blood cells. The mechanism is differentiating, not cytotoxic, and the same dependence on menin exists in NPM1-mutated disease. 📊 Evidence: in AUGMENT-101 (JCO 2025) among 57 evaluable KMT2A-translocated patients complete remission or complete remission with partial haematologic recovery reached 22.8% against a 10% hypothesis (p=0.0036), with an overall response of 63.2% and no detectable residual disease in 15 of 22 responders. In the NPM1 cohort (Blood 2025) the rate was 23.4% (p=0.0014), overall response 46.9%, median remission 4.7 months, and 5 of 30 responders proceeded to transplantation.
⚠️ Prescription-only, prescribed by a haemato-oncology centre. The FDA boxed warning covers differentiation syndrome, which can be fatal and occurred in 25% of patients (33% in KMT2A-translocated myeloid leukaemia), and QTc prolongation with torsades de pointes. Treatment requires a confirmed KMT2A translocation or susceptible NPM1 mutation, is not started with QTcF above 450 msec or white cells above 25 Gi/L, and continues at least six months. In most cases it serves as a bridge to stem cell transplantation rather than an alternative to it.
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