{"entity":{"id":"olutasidenib","kind":"drug","name":"Olutasidenib","aka":[],"tldr":"Olutasidenib is a second IDH1 inhibitor for relapsed AML, with a higher response rate in its pivotal cohort than ivosidenib had in its own.","summary":"Olutasidenib is a selective, allosteric inhibitor of mutant IDH1 that lowers the oncometabolite 2-hydroxyglutarate and lets blocked myeloid cells differentiate. It was approved on 1 December 2022 for relapsed or refractory IDH1-mutated AML, taken twice daily for a minimum of 6 months. In the pivotal cohort (n=147) the CR plus CRh rate was 35% with a median duration of 25.9 months, higher than ivosidenib achieved in its own pivotal cohort, although the two were not compared directly. Differentiation syndrome (16%) carries a boxed warning, and liver enzymes and QT are monitored. Forma Therapeutics developed it and licensed it to Rigel, and combination trials with azacitidine and venetoclax, and a frontline study, are ongoing. It is a second pill for the same genetic subtype of AML, working by switching differentiation back on rather than killing cells.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Olutasidenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Olutasidenib"}],"tags":[],"related":["idh1-r132"],"cancers":["aml","aml-idh"],"sections":[],"technologies":["epigenetic-drugs","idh-inhibitors"],"targets":["idh"],"drugs":[],"companies":["rigel-pharmaceuticals"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome","pivotal-trial"],"trials":["nct06161974"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rezlidhia","modality":"Small-molecule IDH1 inhibitor","mechanism":"Selective, allosteric mutant-IDH1 inhibitor lowering 2-HG.","approvals":[{"region":"US","year":2022,"indication":"Relapsed/refractory IDH1-mutated AML"}],"mechanismSteps":["Binds mutant IDH1 and blocks 2-HG production","Epigenetic demethylation restores differentiation programmes","Blasts mature; responses can take several months"],"dosing":{"route":"Oral","schedule":"150 mg twice daily on an empty stomach, until progression (minimum 6 months)","monitoring":"Differentiation syndrome (boxed warning), hepatotoxicity, QT","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rezlidhia"},"toxicity":[{"event":"Differentiation syndrome","anyGradePct":16,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rezlidhia"},{"event":"Transaminase elevation","anyGradePct":23},{"event":"Nausea","anyGradePct":38}],"access":[],"regulatoryEvents":[{"date":"2022-12-01","type":"approval","region":"US","note":"Study 2102-HEM-101 pivotal cohort"}]},"route":"/drugs/olutasidenib/","neighbours":{"biomarker":[{"id":"idh1-r132","kind":"biomarker","name":"IDH1 R132 mutation","route":"/biomarkers/idh1-r132/"}],"cancer":[{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"idh-mutant-astrocytoma","kind":"cancer","name":"Astrocytoma, IDH-mutant (grades 2 to 4)","route":"/cancers/idh-mutant-astrocytoma/"},{"id":"aml-idh","kind":"cancer","name":"IDH1- and IDH2-mutated acute myeloid leukaemia","route":"/cancers/aml-idh/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"},{"id":"idh-inhibitors","kind":"technology","name":"IDH inhibitors","route":"/technologies/idh-inhibitors/"}],"target":[{"id":"idh","kind":"target","name":"IDH1 / IDH2","route":"/targets/idh/"}],"company":[{"id":"rigel-pharmaceuticals","kind":"company","name":"Rigel Pharmaceuticals","route":"/companies/rigel-pharmaceuticals/"}],"term":[{"id":"differentiation-syndrome","kind":"term","name":"Differentiation syndrome","route":"/terms/differentiation-syndrome/"},{"id":"pivotal-trial","kind":"term","name":"Pivotal (registrational) trial","route":"/terms/pivotal-trial/"}],"trial":[{"id":"nct06161974","kind":"trial","name":"Study of Olutasidenib and Temozolomide in HGG","route":"/trials/nct06161974/"}],"pathway":[{"id":"idh-2hg","kind":"pathway","name":"Mutant IDH / 2-hydroxyglutarate","route":"/pathways/idh-2hg/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}