FDA Approval Summary: Ivosidenib for Treatment of Adult Patients with Relapsed/Refractory Myelodysplastic Syndrome with an IDH1 Mutation

IF 10.2 1区医学 Q1 ONCOLOGY

Clinical Cancer Research Pub Date : 2025-07-01 DOI:10.1158/1078-0432.ccr-25-1005

Ashley C. Woods, E. Dianne Pulte, Xin Wang, Jonathon Vallejo, Ritu Chadda, Nan Zheng, Javier G. Blanco, Sarah E. Dorff, Hongshan Li, Jiang Liu, Olanrewaju O. Okusanya, Richard Pazdur, Marc R. Theoret, R. Angelo de Claro, Kelly J. Norsworthy

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引用次数: 0

Abstract

On October 24th, 2023, the FDA approved a supplemental application for ivosidenib (Tibsovo; Servier) for the treatment of adult patients with relapsed/refractory (R/R) IDH1-mutated myelodysplastic syndrome (MDS). The efficacy of ivosidenib in patients with R/R MDS was established in study AG120-C-001, an open-label, single-arm trial, based on complete remission (CR) + partial remission (PR) rate, duration of CR + PR, and conversion of transfusion dependence (TD) to transfusion independence (TI). With a median follow-up of 27.1 months in 18 patients with IDH1-mutated R/R MDS treated with ivosidenib 500mg once daily, the CR + PR rate was 39% (95% CI: 17.3-64.3) (all CR responses), with a median duration of response not estimable (range 1.9, 80.8+ months). Of the 9 patients that were TD at baseline, 6 achieved TI (67%). Serious adverse reactions in ≥5% included differentiation syndrome, fatigue, and rash. The most common (≥20%) adverse reactions were arthralgia, fatigue, cough, diarrhea, decreased appetite, mucositis, myalgia, and pruritis.

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FDA批准摘要：Ivosidenib用于治疗IDH1突变的复发/难治性骨髓增生异常综合征成人患者

2023年10月24日，FDA批准了ivosidenib (Tibsovo；Servier)用于治疗复发/难治性（R/R） idh1突变骨髓增生异常综合征（MDS）的成人患者。研究AG120-C-001是一项开放标签的单组试验，基于完全缓解(CR) +部分缓解（PR）率、CR + PR持续时间和输血依赖（TD）向输血独立（TI）的转化，确定了ivosidenib对R/R MDS患者的疗效。18例idh1突变的R/R MDS患者接受ivosidenib 500mg每日1次治疗，中位随访27.1个月，CR + PR率为39% (95% CI: 17.3-64.3)（所有CR反应），中位反应持续时间不可估计（范围1.9,80.8+个月）。在基线时为TD的9例患者中，6例达到了TI（67%）。≥5%的严重不良反应包括分化证、疲劳和皮疹。最常见（≥20%）的不良反应是关节痛、疲劳、咳嗽、腹泻、食欲下降、粘膜炎、肌痛和瘙痒。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Cancer Research 医学-肿瘤学

CiteScore

20.10

自引率

1.70%

发文量

1207

审稿时长

2.1 months

期刊介绍： Clinical Cancer Research is a journal focusing on groundbreaking research in cancer, specifically in the areas where the laboratory and the clinic intersect. Our primary interest lies in clinical trials that investigate novel treatments, accompanied by research on pharmacology, molecular alterations, and biomarkers that can predict response or resistance to these treatments. Furthermore, we prioritize laboratory and animal studies that explore new drugs and targeted agents with the potential to advance to clinical trials. We also encourage research on targetable mechanisms of cancer development, progression, and metastasis.