{"title":"Revumenib:首次批准。","authors":"Yahiya Y Syed","doi":"10.1007/s40265-025-02161-5","DOIUrl":null,"url":null,"abstract":"<p><p>Revumenib (Revuforj<sup>®</sup>) is an oral, first-in-class menin inhibitor developed by Syndax Pharmaceuticals for the treatment of KMT2A-rearranged (KMT2Ar) acute leukaemia, NPM1-mutated (NPM1m) acute myeloid leukaemia (AML) and solid tumours. The interaction between menin and the KMT2A protein complex leads to aberrant gene expression, driving leukaemogenic transcription. By blocking this interaction, revumenib promotes differentiation and exerts antileukaemic activity in KMT2Ar acute leukaemias and other menin inhibition-sensitive leukaemias. Revumenib received its first approval on 15 November 2024 in the USA for the treatment of relapsed or refractory (R/R) acute leukaemia with a KMT2A translocation in adult and paediatric patients 1 year and older. This article summarizes the milestones in the development of revumenib leading to this first approval.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"577-583"},"PeriodicalIF":13.0000,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Revumenib: First Approval.\",\"authors\":\"Yahiya Y Syed\",\"doi\":\"10.1007/s40265-025-02161-5\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Revumenib (Revuforj<sup>®</sup>) is an oral, first-in-class menin inhibitor developed by Syndax Pharmaceuticals for the treatment of KMT2A-rearranged (KMT2Ar) acute leukaemia, NPM1-mutated (NPM1m) acute myeloid leukaemia (AML) and solid tumours. The interaction between menin and the KMT2A protein complex leads to aberrant gene expression, driving leukaemogenic transcription. By blocking this interaction, revumenib promotes differentiation and exerts antileukaemic activity in KMT2Ar acute leukaemias and other menin inhibition-sensitive leukaemias. Revumenib received its first approval on 15 November 2024 in the USA for the treatment of relapsed or refractory (R/R) acute leukaemia with a KMT2A translocation in adult and paediatric patients 1 year and older. This article summarizes the milestones in the development of revumenib leading to this first approval.</p>\",\"PeriodicalId\":11482,\"journal\":{\"name\":\"Drugs\",\"volume\":\" \",\"pages\":\"577-583\"},\"PeriodicalIF\":13.0000,\"publicationDate\":\"2025-04-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Drugs\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s40265-025-02161-5\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40265-025-02161-5","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
Revumenib (Revuforj®) is an oral, first-in-class menin inhibitor developed by Syndax Pharmaceuticals for the treatment of KMT2A-rearranged (KMT2Ar) acute leukaemia, NPM1-mutated (NPM1m) acute myeloid leukaemia (AML) and solid tumours. The interaction between menin and the KMT2A protein complex leads to aberrant gene expression, driving leukaemogenic transcription. By blocking this interaction, revumenib promotes differentiation and exerts antileukaemic activity in KMT2Ar acute leukaemias and other menin inhibition-sensitive leukaemias. Revumenib received its first approval on 15 November 2024 in the USA for the treatment of relapsed or refractory (R/R) acute leukaemia with a KMT2A translocation in adult and paediatric patients 1 year and older. This article summarizes the milestones in the development of revumenib leading to this first approval.
期刊介绍:
Drugs is a journal that aims to enhance pharmacotherapy by publishing review and original research articles on key aspects of clinical pharmacology and therapeutics. The journal includes:
Leading/current opinion articles providing an overview of contentious or emerging issues.
Definitive reviews of drugs and drug classes, and their place in disease management.
Therapy in Practice articles including recommendations for specific clinical situations.
High-quality, well designed, original clinical research.
Adis Drug Evaluations reviewing the properties and place in therapy of both newer and established drugs.
AdisInsight Reports summarising development at first global approval.
Moreover, the journal offers additional digital features such as animated abstracts, video abstracts, instructional videos, and podcasts to increase visibility and educational value. Plain language summaries accompany articles to assist readers with some knowledge of the field in understanding important medical advances.