FDA Approval Summary: Vemurafenib for the Treatment of Patients with Erdheim-Chester Disease with the BRAFV600 Mutation.
FDA Approval Summary: Vemurafenib for the Treatment of Patients with Erdheim-Chester Disease with the BRAFV600 Mutation.
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DOI：
10.1634/theoncologist.2018-0295
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发表时间：
2018-12
期刊：
影响因子：
--
通讯作者：
Pazdur R
中科院分区：
文献类型：
--
作者：
Oneal PA;Kwitkowski V;Luo L;Shen YL;Subramaniam S;Shord S;Goldberg KB;McKee AE;Kaminskas E;Farrell A;Pazdur R
FDA已定期批准vemurafenib用于治疗患有BRAFV 600突变的Erdheim-Chester病（ECD）的成人患者。本文描述了FDA对这一罕见患者人群的证据和临床意义的审查。2017年11月6日，美国食品药品监督管理局（FDA）批准vemurafenib用于治疗BRAFV 600突变的Erdheim-Chester病（ECD）成人患者。ECD是一种组织细胞增多症，是一种罕见的疾病，其特征是单核吞噬系统细胞的异常积聚和行为，包括抗原加工细胞、树突状细胞、单核细胞或巨噬细胞。最近发表的数据证实，在ECD患者中BRAFV 600 E突变的频率为54%。批准是基于在VE篮试验（MO 28072）中接受960 mg vemurafenib每日两次的22例患者队列，这是一项单臂、多中心、多队列研究。ECD队列中的患者具有组织学证实的标准治疗难治的具有BRAFV 600突变的ECD。ECD队列的总体缓解率为54.5%（95%置信区间：32.2-75.6），完全缓解率为4.5%。中位随访时间为26.6个月，尚未达到中位缓解时间。ECD队列中最常报告的不良反应（>50%）为关节痛、斑丘疹、脱发、疲乏、心电图QT间期延长和皮肤乳头状瘤。本研究中ECD患者的中位治疗持续时间为14.2个月。本文描述了FDA对vemurafenib用于BRAFV 600突变ECD患者的疗效补充的审查。Vemurafenib是一种靶向BRAF突变的口服单药治疗，是美国食品和药物管理局批准的第一个用于治疗Erdheim-Chester病（ECD）的药物。ECD是一种极为罕见的造血系统肿瘤，代表髓系祖细胞的克隆性增殖。ECD可能累及骨骼和一个或多个器官系统，主要影响50岁和70岁的成年人，男性略占优势。该批准为患有严重和危及生命的疾病的患者提供了一种有效且合理安全的治疗方法，而目前尚无获批的治疗方法。
The FDA has granted regular approval to vemurafenib for the treatment of adult patients with Erdheim‐Chester Disease (ECD) with BRAFV600 mutation. This article describes the FDA review of the evidence and the clinical implications for this rare patient population. On November 6, 2017, the U.S. Food and Drug Administration (FDA) granted regular approval to vemurafenib for the treatment of adult patients with Erdheim‐Chester disease (ECD) with BRAFV600 mutation. ECD is a type of histiocytosis, a rare disorder characterized by an abnormal accumulation and behavior of cells of the mononuclear phagocytic system, which includes antigen‐processing cells, dendritic cells, monocytes, or macrophages. Recently published data confirm a frequency of 54% of BRAFV600E mutations in patients with ECD. Approval was based on a cohort of 22 patients who received 960 mg of vemurafenib twice daily within the VE Basket Trial (MO28072), a single‐arm, multicenter, multiple cohort study. Patients in the ECD cohort had histologically confirmed ECD with BRAFV600 mutations that were refractory to standard therapy. The ECD cohort achieved an overall response rate of 54.5% (95% confidence interval: 32.2–75.6), with a complete response rate of 4.5%. With a median duration of follow‐up of 26.6 months, the median duration of response has not been reached. The most frequently reported adverse reactions (>50%) in the ECD cohort were arthralgia, rash maculo‐papular, alopecia, fatigue, electrocardiogram QT interval prolonged, and skin papilloma. The median treatment duration for ECD patients in this study was 14.2 months. This article describes the FDA review of the vemurafenib efficacy supplement for patients with ECD with BRAFV600 mutations. Vemurafenib, an oral monotherapy targeting a mutation in BRAF, is the first U.S. Food and Drug Administration approval for the treatment of Erdheim‐Chester disease (ECD). ECD is an extremely rare hematopoietic neoplasm that represents clonal proliferation of myeloid progenitor cells. ECD may involve bone and one or more organ systems, primarily affecting adults in their 5th and 7th decades of life, with a slight male predominance. This approval provides an effective and reasonably safe therapy for patients with a serious and life‐threatening condition for which no approved therapy exists.
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