FDA approves axatilimab-csfr for chronic graft-versus-host disease
正文 · 中文译文
FDA批准axatilimab-csfr用于慢性移植物抗宿主病 癌症批准 肿瘤（癌症）/血液系统恶性肿瘤批准通知 进行中 | 癌症加速批准 已验证临床获益 | 癌症加速批准 已撤回 | 癌症加速批准 其他 | 癌症加速批准 2024年8月14日，美国食品药品监督管理局批准axatilimab-csfr（Niktimvo，Incyte公司），一种集落刺激因子-1受体阻断抗体，用于治疗体重至少40 kg的成人和儿童患者在至少接受过两线全身性治疗后失败的慢性移植物抗宿主病（cGVHD）。 Niktimvo的完整处方信息将发布在Drugs@FDA上。 疗效与安全性 疗效在AGAVE-201（NCT04710576）中进行了评估，这是一项随机、开放标签、多中心试验，研究了3个剂量的axatilimab-csfr在复发或难治性cGVHD成人和儿童患者中的效果，这些患者已接受至少2线全身性治疗且需要额外治疗。 主要疗效结局指标为截至第7周期第1天的总缓解率（ORR），其中总缓解包括根据2014年美国国立卫生研究院（NIH）共识发展项目缓解标准判定的完全缓解或部分缓解。在79例接受推荐剂量治疗的患者中，ORR为75%（95% CI：64，84）。首次缓解的中位时间为1.5个月（范围：0.9至5.1）。缓解持续时间的中位数，从首次缓解计算至疾病进展、死亡或针对慢性移植物抗宿主病（GVHD）的新全身治疗，为1.9个月（95% CI：1.6，3.5）。在获得缓解的患者中，自缓解以来至少12个月内，60%（95% CI：43，74）的患者未发生死亡或启动新的全身治疗。 最常见（≥ 15%）的不良反应，包括实验室异常，为天冬氨酸氨基转移酶（AST）升高、感染（病原体未指明）、丙氨酸氨基转移酶（ALT）升高、磷酸盐降低、血红蛋白降低、病毒感染、γ-谷氨酰转移酶（GGT）升高、肌肉骨骼疼痛、脂肪酶升高、疲乏、淀粉酶升高、钙升高、肌酸磷酸激酶（CPK）升高、碱性磷酸酶（ALP）升高、恶心、头痛、腹泻、咳嗽、细菌感染、发热和呼吸困难。 对于体重至少40 kg的患者，axatilimab-csfr的推荐剂量为0.3 mg/kg，最高剂量为35 mg，每2周一次，经30分钟静脉输注给药，直至疾病进展或出现不可接受的毒性。 加速审批程序 本综述使用了评估辅助工具（Assessment Aid），这是申请人自愿提交的材料，旨在便于美国食品药品监督管理局（FDA）进行评估。 本申请获得了优先审评。FDA的加快审评程序在《行业指南：严重疾病药物和生物制品的加快审评程序》中有描述。Axatilimab-csfr被授予孤儿药资格认定和快速通道资格认定，用于治疗慢性移植物抗宿主病（cGVHD）。 医疗专业人员应将所有怀疑与使用任何药物和器械相关的严重不良事件报告至FDA的MedWatch报告系统，或致电1-800-FDA-1088。 如需在研肿瘤产品的单患者IND方面的协助，医疗专业人员可联系OCE的Project Facilitate，电话240-402-0004，或发送电子邮件至OncProjectFacilitate@fda.hhs.gov。 在X上关注肿瘤卓越中心：@FDAOncology。 内容截至日期： 08/14/2024 受监管产品 药物 肿瘤学
正文 · 英文原文
FDA approves axatilimab-csfr for chronic graft-versus-host disease Cancer Approvals Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals On August 14, 2024, the Food and Drug Administration approved axatilimab-csfr (Niktimvo, Incyte Corporation), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Full prescribing information for Niktimvo will be posted on Drugs@FDA. Efficacy and Safety Efficacy was evaluated in AGAVE-201 (NCT04710576), a randomized, open-label, multicenter trial investigating 3 dosages of axatilimab-csfr in adult and pediatric patients with recurrent or refractory cGVHD who had received at least 2 lines of systemic therapy and required additional treatment. The major efficacy outcome measure was overall response rate (ORR) through Cycle 7 Day 1, where overall response included complete response or partial response according to the 2014 NIH Consensus Development Project on Response Criteria. ORR was 75% (95% CI: 64, 84) in the 79 patients treated with the recommended dosage. The median time to first response was 1.5 months (range: 0.9 to 5.1). The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months (95% CI: 1.6, 3.5). In patients who achieved response, no death or new systemic therapy initiation occurred in 60% (95% CI: 43, 74) of patients for at least 12 months since response. The most common (≥ 15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea. The recommended axatilimab-csfr dose in patients who weigh at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every 2 weeks until disease progression or unacceptable toxicity. Expedited Programs This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted priority review. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Axatilimab-csfr was granted Orphan Drug Designation and Fast Track Designation for the treatment of cGVHD. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. Follow the Oncology Center of Excellence on X: @FDAOncology. Content current as of: 08/14/2024 Regulated Product(s) Drugs Oncology
摘要 待译
On August 14, 2024, the Food and Drug Administration approved axatilimab-csfr (Niktimvo, Incyte Corporation), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adul