更新于：2024-05-01

Systemic onset juvenile chronic arthritis

全身型幼年特发性关节炎

更新于：2024-05-01

基本信息

别名

Juvenile RA, Systemic Onset、Juvenile RA, systemic onset、Juvenile arthritis with systemic onset

+ [17]

简介

A category of juvenile idiopathic arthritis defined by the presence of arthritis and high fevers, and accompanied by at least 2 of the following systemic features: lymphadenopathy, organomegaly, rash, or serositis. Macrophage activation syndrome is a well known complication.

关联

项与全身型幼年特发性关节炎相关的药物

Givinostat Hydrochloride

吉维诺司他

靶点

HDACs

作用机制

HDAC抑制剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2024-03-21

Tocilizumab biosimilar(Fresenius Kabi )

托珠单抗生物类似药(Fresenius Kabi )

靶点

IL-6RA

作用机制

IL-6RA拮抗剂

在研机构

Fresenius Kabi Deutschland GmbH [+1]

原研机构

Fresenius Kabi Deutschland GmbH

在研适应症

新型冠状病毒感染 [+6]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

欧盟 [+3]

首次获批日期2023-09-15

Tocilizumab-bavi

托珠单抗生物类似药 (百奥泰生物)

靶点

IL-6RA

作用机制

IL-6RA拮抗剂

在研机构

Biogen Netherlands BV [+2]

原研机构

百奥泰生物制药股份有限公司

在研适应症

多关节型幼年特发性关节炎 [+5]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期2023-01-16

项与全身型幼年特发性关节炎相关的临床试验

CTR20232678 / Recruiting临床3期

一项在1岁至<18岁活动性全身型幼年特发性关节炎受试者中评估乌帕替尼与托珠单抗对照组的有效性、安全性和药代动力学特征的多中心、随机、开放性研究

在1至<18岁活动性全身型幼年特发性关节炎（sJIA）儿童和青少年受试者中评估乌帕替尼与托珠单抗对照组的有效性、安全性和药代动力学特征

开始日期2024-04-08

申办/合作机构

AbbVie, Inc.

[+1]

CTR20231845 / Recruiting临床2期

评估注射用金纳单抗治疗活动性全身型幼年特发性关节炎有效性和安全性的多中心、随机、开放的IIb期临床研究

主要目的：评价注射用金纳单抗在全身型幼年特发性关节炎（sJIA）受试者中的有效性。次要目的：评价注射用金纳单抗在sJIA患者中的安全性；评价注射用金纳单抗的免疫原性。

开始日期2024-01-12

申办/合作机构

长春金赛药业有限责任公司

NCT05609630 / Recruiting临床3期

A Multicenter, Randomized Open-Label Study to Assess the Efficacy, Safety, and Pharmacokinetics of Upadacitinib With a Tocilizumab Reference Arm in Subjects From 1 Year to Less Than 18 Years Old With Active Systemic Juvenile Idiopathic Arthritis

Juvenile Idiopathic Arthritis (JIA) is the most common type of arthritis that affects children. The term "idiopathic" means "of unknown origin". It is a chronic (long-lasting) disease that causes swelling, warmth, and pain of one or more small joints. Systemic JIA ia a rare and serious form of JIA. Systemic" means it may affect not only the joints but other parts of the body, including the liver, lungs and heart. sJIA is more severe and can be more challenging to diagnose and treat than other types of juvenile idiopathic arthritis. It is a lifelong disease for many patients and can continue into adulthood. This study will assess how safe and effective upadacitinib is in treating pediatric and adolescent participants aged 1 to < 18 with systemic juvenile idiopathic arthritis (sJIA) and will include a tocilizumab treatment arm for reference. Adverse events and change in the disease activity will be assessed.
Upadacitinib is an investigational drug being developed for the treatment of sJIA. Participants are assigned to 1 of 2 cohorts. In cohort 1, participants will receive upadacitinib or tocilizumab reference. In cohort 2, participants will receive upadacitinib. Approximately 90 participants with sJIA will be enrolled in approximately 45 sites worldwide.
Participants will receive upadacitinib oral tablets once daily or oral solution twice daily or tocilizumab subcutaneous injection or intravenous infusion as per local label for 52 weeks and followed for approximately 30 days.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits/calls during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.

开始日期2023-10-02

申办/合作机构

AbbVie, Inc.

100 项与全身型幼年特发性关节炎相关的临床结果

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100 项与全身型幼年特发性关节炎相关的转化医学

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0 项与全身型幼年特发性关节炎相关的专利（医药）

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1,177

项与全身型幼年特发性关节炎相关的文献（医药）

2024-02-06·Nature reviews. Rheumatology

Treatment of non-systemic juvenile idiopathic arthritis.

Review

作者: Susan Shenoi ; Gerd Horneff ; Amita Aggarwal ; Angelo Ravelli

In the past two decades, the treatment of juvenile idiopathic arthritis (JIA) has evolved markedly, owing to the availability of a growing number of novel, potent and relatively safe therapeutic agents and the shift of management strategies towards early achievement of disease remission. However, JIA encompasses a heterogeneous group of diseases that require distinct treatment approaches. Furthermore, some old drugs, such as methotrexate, sulfasalazine and intraarticular glucocorticoids, still maintain an important therapeutic role. In the past 5 years, information on the efficacy and safety of drug therapies for JIA has been further enriched through the accomplishment of several randomized controlled trials of newer biologic and synthetic targeted DMARDs. In addition, a more rational therapeutic approach has been fostered by the promulgation of therapeutic recommendations and guidelines. A multinational collaborative effort has led to the development of the recommendations for the treat-to-target strategy in JIA. There is currently increasing interest in establishing the optimal time and modality for discontinuation of treatment in children with JIA who achieve sustained clinical remission. The aim of this Review is to summarize the current evidence and discuss the therapeutic approaches to the management of non-systemic phenotypes of JIA, including oligoarthritis, polyarthritis, enthesitis-related arthritis and psoriatic arthritis.

2024-01-24·Zeitschrift fur Rheumatologie

[Hemophagocytic lymphohistiocytosis and macrophage activation syndrome : A multidisciplinary challenge].

Review

作者: Nikolas Ruffer ; Ricardo Kosch ; Katja Weisel ; Ina Kötter ; Martin Krusche

Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening hyperinflammatory syndrome that is characterized by hyperferritinemia, cytopenia, disseminated intravascular coagulopathy and functional disorders of the liver and the central nervous system. The term macrophage activation syndrome is predominantly used for secondary HLH in the context of autoimmune diseases (e.g., systemic juvenile idiopathic arthritis). In addition, malignancies and genetic inborn errors of immunity can predispose to the development of HLH. Infections (e.g., Epstein-Barr virus) in turn represent possible triggers of an acute episode. Due to the unspecific manifestation of the disease, a systematic evaluation of the organ systems is recommended in the clinical and laboratory analytical clarification of hyperinflammatory syndromes. In general, the treatment should be carried out by a multidisciplinary team with expertise in rheumatology, hematological oncology, infectious diseases and intensive care medicine. The primary treatment of HLH usually consists of glucocorticoids and in cases of a rapid deterioration of the condition anakinra (interleukin 1 block) and intravenous immunoglobulins can be employed. Treatment of the underlying disease should be consequently carried out in parallel, together with antimicrobial treatment.

2024-01-19·Pediatric rheumatology online journal

Establishment and analysis of a novel diagnostic model for systemic juvenile idiopathic arthritis based on machine learning.

Article

作者: Pan Ding ; Yi Du ; Xinyue Jiang ; Huajian Chen ; Li Huang

BACKGROUND:

Systemic juvenile idiopathic arthritis (SJIA) is a form of childhood arthritis with clinical features such as fever, lymphadenopathy, arthritis, rash, and serositis. It seriously affects the growth and development of children and has a high rate of disability and mortality. SJIA may result from genetic, infectious, or autoimmune factors since the precise source of the disease is unknown. Our study aims to develop a genetic-based diagnostic model to explore the identification of SJIA at the genetic level.

METHODS:

The gene expression dataset of peripheral blood mononuclear cell (PBMC) samples from SJIA was collected from the Gene Expression Omnibus (GEO) database. Then, three GEO datasets (GSE11907-GPL96, GSE8650-GPL96 and GSE13501) were merged and used as a training dataset, which included 125 SJIA samples and 92 health samples. GSE7753 was used as a validation dataset. The limma method was used to screen differentially expressed genes (DEGs). Feature selection was performed using Lasso, random forest (RF)-recursive feature elimination (RFE) and RF classifier.

RESULTS:

We finally identified 4 key genes (ALDH1A1, CEACAM1, YBX3 and SLC6A8) that were essential to distinguish SJIA from healthy samples. And we combined the 4 key genes and performed a grid search as well as 10-fold cross-validation with 5 repetitions to finally identify the RF model with optimal mtry. The mean area under the curve (AUC) value for 5-fold cross-validation was greater than 0.95. The model's performance was then assessed once more using the validation dataset, and an AUC value of 0.990 was obtained. All of the above AUC values demonstrated the strong robustness of the SJIA diagnostic model.

CONCLUSIONS:

We successfully developed a new SJIA diagnostic model that can be used for a novel aid in the identification of SJIA. In addition, the identification of 4 key genes that may serve as potential biomarkers for SJIA provides new insights to further understand the mechanisms of SJIA.

项与全身型幼年特发性关节炎相关的新闻（医药）

2024-04-03

·Pharma CMC

爆发！110个新批件发布，30多个品种获批，大批品种过评！

刚刚，NMPA发布2024年04月03日药品批准证明文件送达，本批次共有110个受理号获批，其中21个为一致性评价受理号，过评品种包括：华中药业股份有限公司，维生素B6片国药集团国瑞药业有限公司，注射用更昔洛韦成都天台山制药股份有限公司，注射用维库溴铵杭州中美华东制药有限公司，腺苷注射液等本批次共有46个上市申请受理号获批，30多个品种获准上市，如：辰欣药业的盐酸莫西沙星氯化钠注射液；山东新时代药业的卡培他滨片；江西施美药业的利伐沙班颗粒；成都苑东生物的酒石酸布托啡诺注射液；石家庄四药的头孢呋辛酯干混悬剂；安徽贝克生物制药的利托那韦片；浙江京新药业的吲达帕胺片；浙江诺得药业的替格瑞洛片等。此外，百奥泰生物的托珠单抗注射液适应症获批上市，推测适应症为：全身型幼年特发性关节炎（sJIA）。江苏泰康生物（迈威生物旗下子公司）的地舒单抗注射液新适应获批上市，本次获批适应症尚未披露，此前曾于2023年3月获批用于用于骨折高风险的绝经后妇女的骨质疏松症。华润三九（雅安）药业的3.1类中药新药苓桂术甘颗粒（CDE登记名为苓桂术甘汤颗粒）获批上市。苓桂术甘颗粒主治：温阳化饮，健脾利湿。用于中阳不足之痰饮。症见胸胁支满，目眩心悸，短气而咳，舌苔白滑，脉弦滑。注：黄色标记为一致性评价申请受理号；绿色标记为上市申请受理号

上市批准一致性评价

2024-03-16

·医药观澜

苏庇医药IL-1受体拮抗剂在中国获批新适应症

▎药明康德内容团队报道3月15日，中国国家药监局（NMPA）官网公示，由苏庇医药（Sobi）申报的阿那白滞素注射液新适应症上市申请已获得批准。公开资料显示，这是一种白细胞介素1（IL-1）受体拮抗剂。本次获批的适应症为斯蒂尔病（Still’s disease）。截图来源：NMPA官网斯蒂尔病是一种病因不明的严重罕见炎症性疾病，包括成人斯蒂尔氏病（adult-onset Still’s disease，AOSD）和全身性幼年特发性关节炎（sJIA）。斯蒂尔病潜在致命并发症包括肺动脉高压、急性呼吸衰竭、心肌炎、血栓性血小板减少性紫癜等。斯蒂尔病常用治疗方式包括非甾体类抗炎药（NSAID）、皮质类固醇和改善病情的抗风湿药（DMARD）。研究发现，斯蒂尔病患者常伴有炎症因子的升高，这些炎症因子参与了疾病的发生和发展。研究人员开始针对关键炎性细胞因子开发靶向治疗药物。而针对IL-1的抑制剂可有效控制斯蒂尔病患者的炎症反应，改善症状。根据Sobi公司官网介绍，阿那白滞素（anakinra）正是一种IL-1受体拮抗剂，它能通过与多种组织和器官中表达的IL-1的1型受体结合，阻断IL-1的生物活性，中和这些免疫和炎症过程的关键介质的活性。该药此前已经在中国获批治疗冷吡啉相关周期性综合征（CAPS）、家族性地中海热（FMF）。在全球范围内，阿那白滞素获批的适应症包括CAPS、FMF、类风湿关节炎（RA）、白介素-1受体拮抗剂缺乏症（DIRA）、斯蒂尔病（包括sJIA和成人斯蒂尔病）。据文献报道，在针对斯蒂尔病患者的临床和真实世界研究中，阿那白滞素治疗可使患者达到临床缓解/应答，达成全身和实验室表现的快速、持续改善，患者得以减少或停用皮质类固醇和改善病情抗风湿药物（DMARD）治疗，从而降低与此类治疗相关的药物不良反应风险。参考资料：[1]2024年03月15日药品批准证明文件送达信息发布. Retrieved Mar 15, 2024 from https://www.nmpa.gov.cn/zwfw/sdxx/sdxxyp/yppjfb/20240315151015114.html[2]苏庇医药官网. From https://www.sobi.com/en/pipeline[3]Lyseng-Williamson KA. （2018）Anakinra in Still's disease: a profile of its use. Drugs Ther Perspect. doi: 10.1007/s40267-018-0572-5. 本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

申请上市

2024-03-08

·BioSpace

Fresenius Kabi’s Biosimilar for Actemra Gets FDA Approval

Pictured: Fresenius Kabi's office in Toronto iStock/JHVEPhoto Another biosimilar approval from the FDA was handed down Thursday, as the regulator approved Fresenius Kabi’s Tyenne biosimilar for Genentech’s Acterma (tocilizumab). According to Fresenius Kabi, Tyenne is the first biosimilar to Acterma which has both IV and subcutaneous formulations approved by the FDA. However, as part of a patent settlement with Genentech, the German pharma has a license to market its biosimilar products in the U.S., which will start on confidential license dates. The biosimilar is now approved to treat inflammatory and immune diseases such as rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, giant cell arteritis and systemic juvenile idiopathic arthritis. “The FDA’s approval of our tocilizumab biosimilar is a breakthrough in bringing high-quality, affordable, and accessible autoimmune treatment options to patients and healthcare providers,” Michael Schönhofen, Fresenius Kabi president of biopharma, said in a statement. “We are expanding our biosimilars portfolio for immunology and oncology-related diseases, and we are committed to improving the quality of patients’ lives and lightening the burden on healthcare systems around the world.” The Tyenne biosimilar has already been launched in 10 countries but Fresenius Kabi plans to launch it in additional nations this year and in 2025. Acterma pulled in over CHF 1.2 billion ($1.39 billion) in sales in the U.S. in fiscal year 2023 and CHF 2.6 billion ($2.9 billion) in global sales. “Tyenne met the rigorous FDA requirements for biosimilarity and is another treatment option for patients and their healthcare providers,” Fabrice Romanet, senior vice president of innovation and development at Fresenius Kabi Biopharma, said in a statement. Tyenne is the third FDA-approved biosimilar for Fresenius Kabi as Stimufend, a Neulastat biosimilar, was approved in February 2023 and the Humira biosimilar Idacio was approved in July 2023. The Tyenne approval comes as the FDA gave its approval to two of Sandoz’s biosimilars this week. The regulator approved Sandoz’s Jubbonti, which will go up against Amgen’s Prolia in treating osteoporotic men and postmenopausal women at risk of fracture. The other approved biosimilar is Wyost, similar to Amgen’s Xgeva, which is intended to prevent skeletal-related events in patients with multiple myeloma and solid tumors with bone metastases. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

上市批准专利到期引进/卖出专利侵权