{"entity":{"id":"revumenib","kind":"drug","name":"Revumenib","aka":[],"tldr":"Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.","summary":"Revumenib is a small-molecule menin inhibitor that disrupts the menin-KMT2A interaction, releasing the differentiation block that keeps KMT2A-rearranged and NPM1-mutant leukaemia cells immature. It was approved in 2024, the first menin inhibitor, for relapsed or refractory KMT2A-rearranged acute leukaemia, on AUGMENT-101 (CR/CRh about 23% in heavily pretreated patients), and in 2025 for relapsed or refractory NPM1-mutant AML. Dosing is 270 mg twice daily for patients of 40 kg or more, reduced to 160 mg with strong CYP3A4 inhibitors. Differentiation syndrome and QT prolongation are the key risks. Syndax developed it and ziftomenib (Kura/Kyowa Kirin) was approved in 2025; acquired MEN1 mutations cause resistance, and front-line combinations with venetoclax and azacitidine are being tested. For a newcomer: a pill that makes leukaemia cells grow up instead of multiplying.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Revumenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Revumenib"}],"tags":[],"related":["npm1-mutation"],"cancers":["aml","all-leukemia","aml-npm1-kmt2a"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["menin"],"drugs":[],"companies":["syndax"],"institutions":[],"pathways":["menin-kmt2a"],"terms":[],"trials":["nct07211958"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Revuforj","modality":"Small-molecule menin inhibitor","mechanism":"Disrupts menin-KMT2A interaction, releasing differentiation block.","approvals":[{"region":"US","year":2024,"indication":"Relapsed/refractory KMT2A-rearranged acute leukaemia, age ≥1"},{"region":"US","year":2025,"indication":"Relapsed/refractory NPM1-mutant AML"}],"mechanismSteps":["Revumenib binds menin in the nucleus","Menin can no longer scaffold KMT2A fusion proteins onto chromatin","HOXA9 and MEIS1 leukaemia programmes switch off","Blasts differentiate into mature myeloid cells (differentiation syndrome risk)","Leukaemic clone shrinks"],"dosing":{"route":"Oral","schedule":"270 mg twice daily (≥40 kg) or 160 mg BID with strong CYP3A4 inhibitors; weight-based below 40 kg","modifications":"Hold for QTc >500 ms or grade 3 differentiation syndrome; steroids for differentiation syndrome","monitoring":"ECG before and during treatment; electrolytes; signs of differentiation syndrome","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Differentiation syndrome","note":"AUGMENT-101: ~27% any grade; boxed warning"},{"event":"QTc prolongation"},{"event":"Nausea"},{"event":"Febrile neutropenia"},{"event":"Haemorrhage"},{"event":"Infections"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-12","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11-15","type":"approval","region":"US","note":"Relapsed/refractory KMT2A-rearranged acute leukaemia, age ≥1: first menin inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-10","type":"approval","region":"US","note":"Relapsed/refractory NPM1-mutant AML","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},"route":"/drugs/revumenib/","neighbours":{"biomarker":[{"id":"npm1-mutation","kind":"biomarker","name":"NPM1 mutation","route":"/biomarkers/npm1-mutation/"}],"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"aml-paediatric","kind":"cancer","name":"Acute myeloid leukaemia in children","route":"/cancers/aml-paediatric/"},{"id":"aml-older-unfit","kind":"cancer","name":"Acute myeloid leukaemia in older or unfit patients","route":"/cancers/aml-older-unfit/"},{"id":"mds-higher-risk","kind":"cancer","name":"Higher-risk myelodysplastic syndromes","route":"/cancers/mds-higher-risk/"},{"id":"all-infant","kind":"cancer","name":"Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)","route":"/cancers/all-infant/"},{"id":"aml-npm1-kmt2a","kind":"cancer","name":"NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia","route":"/cancers/aml-npm1-kmt2a/"},{"id":"all-paediatric-relapsed","kind":"cancer","name":"Relapsed and refractory acute lymphoblastic leukaemia in children","route":"/cancers/all-paediatric-relapsed/"},{"id":"aml-secondary","kind":"cancer","name":"Secondary and therapy-related acute myeloid leukaemia","route":"/cancers/aml-secondary/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"},{"id":"menin-inhibitors","kind":"technology","name":"Menin inhibitors","route":"/technologies/menin-inhibitors/"}],"target":[{"id":"hoxa9","kind":"target","name":"HOXA9","route":"/targets/hoxa9/"},{"id":"kmt2a","kind":"target","name":"KMT2A (MLL) rearrangement","route":"/targets/kmt2a/"},{"id":"meis1","kind":"target","name":"MEIS1","route":"/targets/meis1/"},{"id":"menin","kind":"target","name":"Menin","route":"/targets/menin/"},{"id":"npm1","kind":"target","name":"NPM1 mutation","route":"/targets/npm1/"}],"company":[{"id":"syndax","kind":"company","name":"Syndax Pharmaceuticals","route":"/companies/syndax/"}],"pathway":[{"id":"aml-signalling","kind":"pathway","name":"Acute myeloid leukaemia (KEGG map)","route":"/pathways/aml-signalling/"},{"id":"cancer-stem-cells-plasticity","kind":"pathway","name":"Cancer stem cells & phenotypic plasticity","route":"/pathways/cancer-stem-cells-plasticity/"},{"id":"epigenetic-reprogramming","kind":"pathway","name":"Epigenetic reprogramming","route":"/pathways/epigenetic-reprogramming/"},{"id":"menin-kmt2a","kind":"pathway","name":"Menin / KMT2A (HOXA9-MEIS1 axis)","route":"/pathways/menin-kmt2a/"},{"id":"transcription-addiction","kind":"pathway","name":"Transcriptional machinery & addiction","route":"/pathways/transcription-addiction/"}],"trial":[{"id":"augment-101","kind":"trial","name":"AUGMENT-101","route":"/trials/augment-101/"},{"id":"nct07211958","kind":"trial","name":"Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) With a NPM1 Mutation","route":"/trials/nct07211958/"}],"term":[{"id":"cancer-stem-cell-theory","kind":"term","name":"Cancer stem cell theory and phenotypic plasticity","route":"/terms/cancer-stem-cell-theory/"},{"id":"differentiation-syndrome","kind":"term","name":"Differentiation syndrome","route":"/terms/differentiation-syndrome/"},{"id":"epigenetic-progenitor-theory","kind":"term","name":"Epigenetic progenitor theory: cancer without a first mutation","route":"/terms/epigenetic-progenitor-theory/"}],"drug":[{"id":"ziftomenib","kind":"drug","name":"Ziftomenib","route":"/drugs/ziftomenib/"}],"pairing":[{"id":"menin-plus-venetoclax-hma","kind":"pairing","name":"Menin inhibitor + venetoclax + azacitidine","route":"/pairings/menin-plus-venetoclax-hma/"}],"idea":[{"id":"idea-menin-infant-all","kind":"idea","name":"Menin inhibitors for infant KMT2A-rearranged ALL","route":"/ideas/idea-menin-infant-all/"}],"person":[{"id":"eunice-wang","kind":"person","name":"Eunice S. Wang","route":"/people/eunice-wang/"},{"id":"eytan-stein","kind":"person","name":"Eytan M. Stein","route":"/people/eytan-stein/"},{"id":"ghayas-issa","kind":"person","name":"Ghayas C. Issa","route":"/people/ghayas-issa/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-undruggable-targets","kind":"bottleneck","name":"The undruggable drivers","route":"/bottlenecks/b-undruggable-targets/"}],"paper":[{"id":"paper-augment-101-revumenib-menin-nature-2023","kind":"paper","name":"AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation","route":"/key-papers/paper-augment-101-revumenib-menin-nature-2023/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}