A Phase 3, Open-Label, Extension Study to Evaluate The Long-Term Safety And Efficacy of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

The purpose of the study is to assess the long-term safety and efficacy of rozanolixizumab in adult study participants with ocular myasthenia gravis.

开始日期2026-07-27

申办/合作机构

UCB Biopharma SRL

NCT07570589

/ Not yet recruitingN/AIIT

An Observational, Prospective, Multicenter, Open-label Study to Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)

Generalized Myasthenia Gravis (gMG) is a rare autoimmune disease (a disease in which the body attacks its own tissues) that causes muscle weakness and significant fatigue.
Current treatments (corticosteroids, plasma exchange, intravenous immunoglobulin infusions) improve symptoms in many patients. However, many continue to suffer from fatigue and fatigability that are not well measured by standard tools. Moreover, these treatments can cause significant long-term side effects, reducing quality of life.
New treatments such as Rozanolixizumab (ROZ) are now available. They act rapidly and are well tolerated, allowing better symptom control while reducing the risks associated with conventional treatments.
To properly evaluate these new treatments, it is essential to understand patients' perspectives on their effectiveness. The scales used by physicians do not always capture all the symptoms experienced by patients, particularly fatigability. This is why a new tool has been developed: the MG symptoms PRO. This questionnaire allows patients to assess their own symptoms (fatigue, weakness of the eyes, mouth, breathing, muscle fatigability) in detail.
This research aims to better understand the effectiveness of treatments from the patients' perspective in order to improve their care.
The goal of the study is to evaluate the impact of Rozanolixizumab administration in real-world practice through the MG symptoms PRO questionnaire. This is an observational study, meaning that the medication is prescribed by the physician according to current regulations, and the study simply collects routine medical data during your follow-up, over a period of approximately 9 months.

开始日期2026-06-01

申办/合作机构

Centre Hospitalier Universitaire de Nice

NCT07463521

/ Not yet recruiting临床3期

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis.

开始日期2026-05-29

申办/合作机构

UCB Biopharma SRL

100 项与罗泽利昔单抗相关的临床结果

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100 项与罗泽利昔单抗相关的转化医学

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100 项与罗泽利昔单抗相关的专利（医药）

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项与罗泽利昔单抗相关的文献（医药）

2026-04-01·ADVANCES IN THERAPY

Assessing the Value Contribution of Vyvgart® (Efgartigimod Alfa) in the Treatment of Generalized Myasthenia Gravis with Acetylcholine Receptor Antibody in Spain Through Multi-criteria Decision Analysis

Article

作者: Gil, Alicia ; Toja-Camba, Francisco J ; Martínez, Eva ; Díaz, Carmina ; Serra, José M ; Trillo, Jose L ; Martín Machín, Celia ; Guerrero, Antonio ; Abad, María R ; Cortés-Vicente, Elena

INTRODUCTION:

Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder that currently lacks a curative treatment. Efgartigimod alfa is the first human IgG1 Fc fragment approved in Spain for the management of this condition. This study aims to evaluate the value of efgartigimod for treating gMG with anti-acetylcholine receptor (gMG AChR+) antibodies compared with ravulizumab, zilucoplan, and rozanolixizumab using a multi-criteria decision analysis (MCDA) framework.

METHODS:

A multidisciplinary group of eight experts evaluated the value of efgartigimod against ravulizumab, zilucoplan, and rozanolixizumab. The MCDA framework adapted for evaluating orphan drugs (ODs) in Spain, comprising nine quantitative and three qualitative criteria, was used.

RESULTS:

gMG AChR+ has been recognised as a severe and debilitating condition with considerable unmet needs. Efgartigimod achieved favourable average scores compared with ravulizumab, zilucoplan, and rozanolixizumab across all comparative parameters, including efficacy, safety, and patient-reported outcomes (PROs), potentially resulting in cost savings. Efgartigimod was deemed to have a significant therapeutic impact, with supporting data considered high quality. Efgartigimod alfa showed a higher overall value contribution than the three comparators, with the difference being most notable for ravulizumab. Furthermore, it was concluded that efgartigimod aligns well with the specific priorities, objectives, and capacities of the National Healthcare System (NHS).

CONCLUSION:

Efgartigimod has been recognised as a valuable option for gMG AChR+ treatment in Spain by a multidisciplinary panel of experts through the application of MCDA, receiving higher scores compared with ravulizumab, zilucoplan, and rozanolixizumab.

2026-04-01·JOURNAL OF NEUROLOGY

Efficacy and safety of complement inhibitors and FcRn blockers in generalized AChR antibody-positive myasthenia gravis: a meta-analysis

Review

作者: Di Giovanni, Simone ; Filippakopoulou, Eugenia ; Arnaoutoglou, Marianthi ; Kimiskidis, Vasilios ; Gavriilaki, Maria

BACKGROUND:

Antibody status is increasingly used to inform treatment strategies in generalized myasthenia gravis (gMG). We evaluated the efficacy and safety of complement inhibitors and neonatal Fc receptor (FcRn) blockers versus placebo or standard care in adults with acetylcholine receptor antibody-positive (AChR-Ab⁺) gMG.

METHODS:

We systematically searched MEDLINE, EMBASE, Cochrane Central Register Controlled Trials, and ClinicalTrials.gov through November 2024. Eligible randomized controlled trials (RCTs) informed short-term analyses while long-term outcomes were extracted from open-label extension (OLE) studies. Pooled mean difference (MD) and odds ratio (OR) were calculated using random-effects model.

RESULTS:

Six RCTs (n = 739) and four OLEs (n = 588) evaluating eculizumab, ravulizumab, zilucoplan, efgartigimod, rozanolixizumab, and batoclimab were included. Both drug classes significantly improved mean changes from baseline versus placebo in Myasthenia Gravis Activities of Daily Living [MD 1.7, 95% confidence interval (CI) 1.1-2.3], Quantitative Myasthenia Gravis [MD 2.7, 95%(CI)1.8-3.5], Myasthenia Gravis Composite [MD 6.3, 95%(CI) 5-7.6], MGQoL15r [MD 3.4, 95%(CI)1.2-5.6], and Neuro-QoL [MD 4.5, 95%(CI)1.2-7.7]. Odds of achieving clinically meaningful MG-ADL and QMG improvements were more than doubled (ORs 2.7 and 3.5). Risks of clinical worsening and rescue therapy use were reduced by 72% and 48%, respectively. Complement inhibitors OLEs showed durable benefit up to 156 weeks; 30% of patients reduced corticosteroid doses. Rates of serious adverse events, discontinuation, and mortality were comparable to placebo.

CONCLUSION:

In AChR-Ab⁺ gMG, complement inhibitors and FcRn blockers yield clinically meaningful improvements with favourable safety profiles. Complement inhibition additionally confers sustained benefits and corticosteroid-sparing effects in this population over long-term.

PROTOCOL REGISTRATION:

PROSPERO ID: CRD42024513406.

2026-03-01·NAUNYN-SCHMIEDEBERGS ARCHIVES OF PHARMACOLOGY

Pharmacological and speech-language pathology management of dysphagia in patients with myasthenia gravis

Review

作者: Ferrara, F ; De Berardinis, F

Myasthenia gravis (MG) is a rare, complex autoimmune neuromuscular disorder characterized by fluctuating muscle weakness. Bulbar involvement, particularly dysphagia (difficulty swallowing), is a common and debilitating symptom that significantly increases the risk of aspiration pneumonia, malnutrition, and dehydration, profoundly impacting quality of life. The management of myasthenic dysphagia requires a sophisticated, interdisciplinary approach. This review aims to provide a comprehensive overview of the current and emerging pharmacological treatments for myasthenia gravis and to detail the specific assessment and management strategies employed by speech-language pathologists (SLPs) for associated dysphagia. A narrative review of the literature was conducted, focusing on the pathophysiology, classification, and treatment of MG, with a specific emphasis on bulbar dysfunction. The review synthesizes evidence for traditional immunosuppressants, novel targeted biologics (complement inhibitors and FcRn antagonists), and evidence-based speech-language pathology interventions. Pharmacological management has evolved from symptomatic treatment (anticholinesterases) and broad immunosuppression (corticosteroids, azathioprine) to highly specific, targeted therapies. Complement inhibitors (e.g., eculizumab, ravulizumab) and neonatal Fc receptor (FcRn) antagonists (e.g., efgartigimod, rozanolixizumab) have demonstrated significant and rapid efficacy in reducing autoantibody load and improving clinical scores (MG-ADL, QMG) in generalized MG, including for patients with refractory disease. Concurrently, SLP management is critical for functional safety and efficacy. This includes precise assessment using clinical (e.g., EAT-10, bedside swallowing evaluations) and instrumental methods (VFSS, FEES) to characterize the pathophysiology of weakness and fatigability. Management is individualized, focusing on compensatory strategies (e.g., postural adjustments, diet modification standardized by the IDDSI framework) and behavioral techniques, all timed to coincide with peak medication efficacy. The treatment landscape for MG has been revolutionized by targeted biologics, offering profound improvements for many patients. Effective management of dysphagia, however, relies on the synergy between optimal medical therapy and specialized SLP intervention. This integrated approach is essential to ensure swallowing safety, nutritional adequacy, and improved quality of life for individuals living with myasthenia gravis.

259

项与罗泽利昔单抗相关的新闻（医药）

2026-05-21

·美通社

强生旗下安力威®（尼卡利单抗注射液）在华获批，新型FcRn拮抗剂为广泛全身型重症肌无力患者群体带来持续平稳的疾病控制

中国首个且目前唯一 [1,2,3,4] 获批用于治疗 12 岁及以上青少年和成人自身抗体（ AChR 或 MuSK ）阳性的全身型重症肌无力（ gMG ）的 FcRn 拮抗剂在成人和青少年患者的关键性临床研究中，安力威 ® 能快速且显著降低免疫球蛋白 G （ IgG ）水平 —— 该抗体是导致重症肌无力的根本原因之一 [5,6] 在 Vivacity-MG3 关键性研究及正在进行的开放标签扩展期，接受安力威 ® 治疗的全身型重症肌无力患者实现了长达 20 个月的持续平稳疾病控制和症状改善 [5,7] 北京 2026年5月21日 /美通社/ -- 强生公司今日宣布，公司旗下全人源新生儿Fc受体（FcRn）拮抗剂安力威 ® （尼卡利单抗注射液）已获国家药品监督管理局批准，联合常规治疗药物用于治疗全身型重症肌无力。此次获批为广泛的全身型重症肌无力患者群体（抗乙酰胆碱受体（AChR）抗体阳性或抗肌肉特异性受体酪氨酸激酶（MuSK）抗体阳性的12岁及以上青少年和成人）提供了新的治疗选择，实现持续平稳的疾病控制 [5,6,7] 。该新药上市申请获得了国家药品监督管理局药品审评中心的优先审评审批资格 [8] ，同时安力威 ® 也是全国首个获批开展原液境内分段生产试点的进口生物制品，该药品原液在国内生产，制剂和包装在境外生产 [9] 。全身型重症肌无力（gMG）是一种慢性、可致残性自身免疫性疾病，目前针对该疾病仍存在未满足的临床需求，需要能提供持续平稳疾病控制、且具有明确安全性的有效疗法 [5,6,10,11] 。抗AChR及抗MuSK抗体阳性患者占全部抗体阳性gMG患者的90%及以上 [5] 。安力威 ® 是一种FcRn拮抗剂，其作用机制可显著降低免疫球蛋白G（IgG）（包括有害的IgG自身抗体）水平，同时不对其他适应性免疫和先天免疫功能产生可检测的额外影响 [5] 。此次批准基于Vivacity-MG3关键性研究的数据。在所有针对成人gMG的FcRn拮抗剂注册临床试验中，该研究设置了迄今为止最长的主要终点。研究亮点包括 [1-5,11,12] ：以MG-ADL评分改善为衡量标准，在整个24周治疗期间，安力威 ® 联合标准治疗相比安慰剂联合标准治疗展现出更优异的疾病控制效果。这使得患者能够改善包括咀嚼、吞咽、言语及呼吸等基本日常功能。在正在进行的gMG开放标签扩展期研究中，接受安力威 ® 联合标准治疗的患者在长达20个月的随访期内持续保持症状改善。安力威 ® 展现出快速且持续平稳的自身抗体水平降低效果：首次给药两周后，总IgG抗体水平最高降低约75%，并在24周内持续维持。目前正在进行的针对抗AChR及抗MuSK抗体阳性、12至17岁青少年患者的2/3期研究 Vibrance-MG 结果显示，安力威 ® 联合标准治疗达到了主要终点：在24周治疗期间血清总IgG水平降低约69%，同时在MG-ADL a 和QMG b 量表改善方面达到次要终点。 [6] 安力威 ® 在 Vivacity-MG3 研究和正在进行的 Vibrance-MG 研究中，展现出一致的安全性特征，在成人与青少年患者中的耐受性相当。 [5,6,13] 强生创新制药中国区总裁Cherry Huang表示："安力威 ® 的上市，标志着强生在推动罕见病治疗进步方面迈出了关键一步。该药物为广泛的重症肌无力患者群体带来了持续平稳的疾病控制，有望提升患者的治疗体验，帮助他们重拾生活主动权。这一重要里程碑的实现，得益于强生多年来对尼卡利单抗的深入科学探索、跨界协同创新以及始终如一的坚定信念。我们期待这一创新疗法能够尽快惠及更多中国患者，助力他们重获健康与希望。" 2025年4月，美国食品药品监督管理局首次批准安力威 ® 用于治疗gMG患者 [14] ；同年9月，巴西和日本卫生监管机构相继批准该疗法 [15,16] ；12月，欧洲委员会授予其在欧盟的上市许可 [15,16] 。目前，全球多个卫生监管机构正在审评安力威 ® 治疗gMG的上市申请。备注： MG-ADL（重症肌无力日常生活活动量表）用于在临床上快速评估影响患者日常生活活动的症状，评分范围为0至24分，分值越高表示症状越严重。 [ 17] QMG（重症肌无力定量量表）共包含13项内容，由临床医生实施评估，用于量化重症肌无力的疾病严重程度，总分范围为0至39分，分值越高表示疾病越严重。 [ 17] 关于全身型重症肌无力（ gMG ）重症肌无力（MG）是一种自身免疫性疾病，患者的免疫系统错误地产生抗体（如抗乙酰胆碱受体[AChR]抗体、抗肌肉特异性受体酪氨酸激酶 [MuSK]抗体），这些抗体可作用于神经-肌肉接头处，阻断或干扰神经向肌肉的正常信号传导，从而影响或阻止肌肉收缩 [ 4] 。全球约有 70万名MG患者 [ 18] 。MG可发生于男女各年龄段及各种族和族群，但多在年轻女性和老年男性中发病 [ 19] 。 MG的早期症状通常以眼部表现为主，但约85%的患者会进展为全身型重症肌无力（gMG），可表现为严重肌无力，并可能伴随言语和吞咽困难等症状。 [ 4,17] 关于 3 期 VIVACITY-MG3 研究 [ 5] 3期Vivacity-MG3研究（NCT04951622）旨在评估在gMG这一不可预测、患者未满足需求仍高的慢性疾病中，持续给药下的疗效与安全性。研究纳入了对现行标准治疗（SOC）应答不足（MG-ADL ≥6）的成人gMG患者，不论抗体阳性或阴性，共入组199名患者，其中153名为抗体阳性患者，参与为期24周的双盲安慰剂对照试验。患者按1:1随机分配至尼卡利单抗联合目前SOC组（30 mg/kg静脉注射负荷剂量，随后每两周15 mg/kg）或安慰剂联合目前SOC组。基线人口学特征在两组间平衡（尼卡利单抗组77例，安慰剂组76例）。主要疗效终点为比较两组MG-ADL总评分从基线至第22、23及24周的平均变化情况。关键次要终点包括QMG评分的变化。此外，正在进行的开放标签扩展期（OLE）研究将对长期疗效与安全性开展持续评估。关于 2/3 期 Vibrance-MG 研究 [ 6,20] 2/3期Vibrance-MG研究（NCT05265273）是一项正在进行的开放标签研究，旨在评估尼卡利单抗对2至＜12岁儿童及12至＜18岁青少年gMG患者的疗效。研究纳入了7名12-17岁gMG患者，筛查时MGFA分型为II-IV，并对目前稳定SOC治疗应答不足。入组患者须呈抗AChR或抗MuSK抗体阳性。研究包括最长四周的筛查期、24周开放标签积极治疗期（期间患者每两周静脉注射尼卡利单抗）以及长期扩展期；最后一次给药后8周将进行安全性随访评估。主要终点为24周内尼卡利单抗对青少年gMG患者总血清IgG的影响、药物安全性及耐受性。次要终点包括24周内MG-ADL及QMG评分的变化。关于安力威 ® （尼卡利单抗注射液）安力威 ® （尼卡利单抗注射液）是一种单克隆抗体，旨在以高亲和力结合并阻断 FcRn，从而降低循环中的免疫球蛋白 G（IgG）水平，同时不对其他适应性和先天性免疫功能产生可检测的额外影响 [ 5] 。这些IgG抗体是全身型重症肌无力（gMG）的主要致病因素 [ 4] 。安力威 ® 已在中国获批联合常规治疗药物，用于治疗抗乙酰胆碱受体（AChR）抗体阳性或抗肌肉特异性受体酪氨酸激酶（MuSK）抗体阳性的成人及12岁及以上青少年gMG患者。尼卡利单抗正在自身抗体相关疾病领域的三个关键方向开展进一步研究，包括罕见自身抗体疾病、母体同种抗体介导的母胎疾病以及风湿性疾病。 [ 21-29] 该在研单克隆抗体旨在以高亲和力结合并阻断FcRn，从而降低循环中的自身及同种IgG抗体水平，同时不对其他适应性和先天性免疫功能产生可检测的额外影响 [ 30] 。美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）已授予尼卡利单抗多项重要资格认定 [ 14] ，包括： 2019年10月，欧洲EMA授予胎儿及新生儿溶血病（HDFN）适应症孤儿药资格 2019年7月，美国FDA授予胎儿及新生儿溶血病（HDFN）适应症及温抗体型自身免疫性溶血性贫血（wAIHA）适应症快速通道认定；2021年12月授予全身型重症肌无力（gMG）适应症快速通道认定；2024年3月授予胎儿及新生儿同种免疫性血小板减少症（FNAIT）适应症快速通道认定；2025年3月授予干燥综合征（SjD）适应症快速通道认定 2019年12月，美国FDA授予wAIHA适应症孤儿药资格；2020年6月授予HDFN适应症孤儿药资格；2021年2月授予gMG适应症孤儿药资格；2021年10月授予慢性炎性脱髓鞘性多发性神经根神经病（CIDP）适应症孤儿药资格；2023年12月授予FNAIT适应症孤儿药资格 2024年2月，美国FDA授予HDFN适应症突破性疗法认定；2024年11月授予干燥综合征适应症突破性疗法认定 2024年第四季度，美国FDA授予gMG适应症优先审评资格参考文献 https://cn.zailaboratory.com/products/ https://www.ucbchina.com/About-UCB/%E4%B8%BB%E8%A6%81%E4%BA%A7%E5%93%81 https://www.cde.org.cn/main/xxgk/listpage/2f78f372d351c6851af7431c7710a731 Neurology Branch of the Chinese Medical Association, Neuroimmunology Group. Chinese Journal of Neurology, 2025; 58(7): 721–741. Antozzi, C et al., Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study. Lancet Neurol. 2025 Feb;24(2):105-116. doi: 10.1016/S1474-4422(24)00498-8. Strober J et al. Safety and effectiveness of nipocalimab in adolescent participants in the open label Phase 2/3 Vibrance-MG clinical study. Presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting. October 2024. Antozzi, C et al., Long-Term Safety and Efficacy of Nipocalimab in Generalized Myasthenia Gravis: Vivacity-MG3 Open-Label Extension Phase Results. Abstract #022 for poster presentation at 2025 American Academy of Neurology Congress. April 2025 https://www.cde.org.cn/main/xxgk/listpage/2f78f372d351c6851af7431c7710a731 https://m.bjnews.com.cn/detail/1747748925129673.html Law N, et al. The Lived Experience of Myasthenia Gravis: A Patient-Led Analysis. Neurol Ther. 2021, 10(2):1103-1125. doi: 10.1007/s40120-021-00285-w. Epub 2021 Oct 23. Vera Bril, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023; 22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. James F Howard Jr, et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021; 20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. IMAAVY TM U.S. Prescribing Information. https://www.jnj.com/media-center/press-releases/johnson-johnson-receives-fda-approval-for-imaavytm-nipocalimab-aahu-a-new-fcrn-blocker-offering-long-lasting-disease-control-in-the-broadest-population-of-people-living-with-generalized-myasthenia-gravis-gmg https://www.jnj.com/media-center/press-releases/johnson-johnson-receives-european-commission-approval-of-imaavy-nipocalimab-a-new-fcrn-blocker-offering-sustained-disease-control-in-a-broad-population-of-people-living-with-generalised-myasthenia-gravis-gmg Carlo Antozzi & Marie Fitzgibbon (2025) An evaluation of nipocalimab for the treatment of generalized myasthenia gravis, Expert Opinion on Biological Therapy, 25:10, 1047-1058, DOI: 10.1080/14712598.2025.2561935 Wolfe GI, et al. Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. Chen J, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. 2020; 27:5:100063. doi: 10.1016/j.lanwpc.2020.100063. Dresser, Laura et al. Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. J Clin Med. 2021 May 21;10(11):2235. doi: 10.3390/jcm10112235. ClinicalTrials.gov. NCT05265273. Available at: https://clinicaltrials.gov/study/NCT05265273 . Last accessed: February, 2026 ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT06449651. Available at: https://clinicaltrials.gov/study/NCT06449651 . Last accessed: February, 2026. ClinicalTrials.gov Identifier: NCT06533098 Available at: https://clinicaltrials.gov/study/NCT06533098 . Last accessed: February, 2026. Seth, N. P., et al. Nipocalimab, an immunoselective FcRn blocker that lowers IgG and has unique molecular properties. MAbs. 2025;17(1):2461191. doi: 10.1080/19420862.2025.2461191. Epub 2025 Feb 12. 关于强生创新制药<\/b><\/p> 作为首批进入中国的跨国制药企业之一，强生创新制药自1985年在华成立以来，始终致力于以科学领拓医药未来。科学为本，我们是忠于科学的创新者，积极探索突破性疗法，变革健康未来；使命为上，我们是健康中国的贡献者，以应对中国最为迫切的医药健康需求为己任，共筑健康中国；患者为先，我们是心系患者的同行者，不断创新治疗途径，帮助患者寻找治疗方法抵御疾病。作为强生在华制药子公司，我们聚焦于肿瘤学、免疫学、神经科学、心肺疾病、眼科等能够为人类健康带来巨大改变的疾病领域。<\/p>

2026-05-21

·丁香园 Insight 数据库

65 亿美元收购！强生重磅新药在中国获批上市

5 月 21 日，强生宣布「尼卡利单抗」在国内首次获批上市，用于治疗自身抗体阳性的全身型重症肌无力（gMG）成人患者和大于 12 岁的青少年患者。此前，该产品曾被 CDE 纳入优先审评。截图来源：强生官微尼卡利单抗（Nipocalimab）是一种 FcRn 抗体。与艾加莫德和罗泽利昔珠单抗同属于 FcRn 拮抗剂不同，该药属于 FcRn 阻断剂，以高亲和力结合 FcRn，阻断其与免疫球蛋白 G (IgG) 抗体的结合，减少 IgG 抗体的回收和循环，从根本上降低血液中自身抗体水平，同时保持免疫功能而不引起广泛的免疫抑制。截图来自：强生官方资料 2020 年 8 月，强生以 65 亿美元收购 Momenta Pharmaceuticals，囊获这款重磅新药。2025 年 4 月该药首次在美国获批上市，成为当时首个且唯一获批用于治疗抗 AChR 抗体和抗 MuSK 抗体阳性成人及 12 岁及以上儿童 gMG 患者的 FcRn 阻断剂，而后又陆续在欧盟、日本、英国获批上市。权益流转情况（上）全球批准历史（下）截图来自：Insight 数据库网页版彼时 FDA 对尼卡利单抗的批准是基于 III 期临床试验 Vivacity-MG3 的积极结果（试验登记号：NCT04951622/CTR20212693）。这是一项随机、双盲、安慰剂对照 III 期临床研究，入组患者为对当前标准疗法（SOC）反应不佳的抗体阳性成人 gMG 患者。主要终点是抗体阳性患者在治疗第 22、23 和 24 周内重症肌无力日常活动评分（MG-ADL）相对于基线的变化，次要终点包括重症肌无力定量评分 (QMG) 等。关键研究历史时光轴截图来自：Insight 数据库网页版关键临床结果显示：在治疗第 22、23 和 24 周时，接受「尼卡利单抗+SOC」的患者 MG-ADL 评分改善了 4.70 分，显著高于「安慰剂+SOC」组的 3.25 分改善（P=0.002）；在次要终点 QMG 方面，与安慰剂+SOC 相比，尼卡利单抗+SOC 的 QMG 评分改善 4.86 分（vs 安慰剂组 2.05 分），患者不同肌肉群的力量和功能得到显著改善（P<0.001）；尼卡利单抗+SOC 组 MG-ADL 评分改善 ≥2 分的比例为 68.8%，而安慰剂+SOC 组这一数值为 52.6%（P=0.021），进一步表明使用尼卡利单抗治疗可减轻 gMG 对患者日常生活的影响。安全性方面，尼卡利单抗的耐受性总体良好，不良事件的总体发生率与安慰剂+SOC 组相似。尼卡利单抗治疗 gMG 还具有长期的有效性和安全性。正在进行的 III 期开放标签扩展研究（OLE）的结果显示，接受尼卡利单抗联合标准治疗的 gMG 患者在长达 20 个月的随访中仍然保持了良好的状态。同时，强生还正在开展一项针对 12～17 岁抗 AChR 和抗 MuSK 抗体阳性的青少年 gMG 患者的 II/III 期儿科研究（Vibrance-MG）。这项研究数据显示，尼卡利单抗联合 SOC 疗法达到了其主要终点，即 24 周内血清总 IgG 水平降低 69%，次要终点 MG-ADL 和 QMGc 评分均得到改善。重症肌无力是一种罕见的、慢性自身免疫性疾病。免疫系统会错误地攻击神经肌肉接头处的蛋白质，产生抗体（例如抗乙酰胆碱受体 [AChR]、抗肌肉特异性酪氨酸激酶 [MuSK] 或抗低密度脂蛋白相关蛋白 4 [LRP4]），这些抗体会阻碍或破坏正常功能，阻止信号从神经传递到肌肉。在中国，gMG 已被纳入《第一批罕见病目录》。据弗若斯特沙利文报告，全球肌无力患者约 120 万人，其中中国患者约 22 万人，存在巨大未满足临床需求。近年来，生物制剂的出现为这一疾病领域带来了新希望，尤其是 C5 补体抑制剂和 FcRn 单抗在此领域更是活跃。结语据 Insight 数据库统计，当前全球有超 60 款针对重症肌无力的生物药（仅统计积极状态）已经进入临床开发。而仅仅国内此前就已有 7 款获批上市：阿斯利康的两代 C5 补体抑制剂「依库珠单抗」和「瑞利珠单抗」； Argenx/再鼎医药的 FcRn 单抗「艾加莫德」及其皮下注射剂；优时比的 FcRn 单抗「罗泽利昔珠单抗」、C5 补体抑制剂「泽卢克布仑钠」；荣昌生物的 BLyS/APRIL 双靶点融合蛋白「泰它西普」；翰森制药引进的 CD19 单抗「伊奈利珠单抗」。其中 5 款都是在 2025 年及以后新获批的，可以说这两年是国内重症肌无力新药的「爆发之年」了。而申报上市的新药中还包括石药集团/和铂医药的 FcRn 单抗「巴托利单抗」以及阿斯利康的 C5/Albumin 纳米双抗「戈鲁利单抗」。可以看出，重症肌无力治疗药物目前以 C5 和 FcRn 靶点为主，各大药企围绕该疾病进行了深入且差异化的布局：阿斯利康针对 C5 补体进行迭代纵深布局；优时比双机制布局，有望探索双药联用方案；Argenx/再鼎作为同类首款，最先推进皮下注射剂开发；荣昌生物以创新机制破局。而在临床开发产品中，CD19、CD20 单抗开始频频出现，靶向 CD19 和 BCMA 的 CAR-T 疗法也颇具前景。对于强生而言，自免是该公司制药板块的关键业务领域，依据其财报显示，2025 年度自免领域为强生创收 157.28 亿美元。不过如今因专利到期和强敌环伺，强生在该领域的营收已经连续两年出现了下滑。因此，本次获批的尼卡利单抗也是强生寄予厚望的未来支柱之一。除了 gMG，强生还正在临床中探索该药治疗多种自免疾病的效果，包括特发性炎症性肌病、干燥综合征、类风湿关节炎、新生儿溶血性疾病、自身免疫性溶血性贫血、慢性炎性脱髓鞘性多发性神经病等。扫描二维码添加「小音」，回复口令新药获批，即可获得 Insight 整理的 2026 年全球新药获批上市资源大礼包👇（每月初更新一次，目前更新至 4 月）。封面来源：企业 Logo 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：ccai PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

2026-05-21

·allsci.com

Immunovant posts hopeful data for FcRn inhibitor in rheumatoid arthritis post batoclimab failure

Immunovant reported preliminary Week 16 data for its FcRn inhibitor IMVT-1402 in heavily pretreated rheumatoid arthritis patients, with ACR20, ACR50, and ACR70 response rates of 72.7%, 54.5%, and 35.8%, respectively, according to results disclosed alongside the company’s fiscal year 2026 financial results. The data arrive weeks after Immunovant discontinued development of batoclimab following failed Phase III thyroid eye disease studies, increasing investor focus on IMVT-1402 as the company’s lead FcRn inhibitor franchise. The open-label study enrolled adults with difficult-to-treat rheumatoid arthritis who had failed at least two prior advanced therapies, including biologic and targeted synthetic DMARDs. Among 165 evaluable participants, response rates remained consistent in a subgroup previously treated with both JAK and anti-TNF inhibitors. The IMVT-1402 rheumatoid arthritis study is a two-period trial enrolling heavily pretreated adults with difficult-to-treat rheumatoid arthritis who have failed at least two prior mechanisms of advanced therapy, including biologic and targeted synthetic DMARDs. Period 1 is an open-label, 16-week phase in which all 170 enrolled participants received IMVT-1402 600 mg weekly by subcutaneous injection. Of the 170 enrolled, 165 were evaluable at Week 16. The primary endpoint for the trial is the proportion of Period 1 ACR20 responders who maintain that response at Week 28. At Week 16, the observed ACR20, ACR50, and ACR70 response rates across the 165 evaluable participants were 72.7%, 54.5%, and 35.8%, respectively, with participants who discontinued before Week 16 counted as non-responders. Within the subset of 107 participants who had failed at least one JAK inhibitor and at least one anti-TNF inhibitor — a group representing a particularly refractory population — the ACR20, ACR50, and ACR70 rates were 72.0%, 53.3%, and 37.4%, respectively, showing minimal attenuation compared to the overall cohort. IMVT-1402 was described as safe and well-tolerated, with no new drug-related safety signals identified during Period 1. These are preliminary, open-label data from a single-arm period, and the absence of a concurrent placebo arm limits direct causal interpretation. The randomized Period 2, with topline data expected in the H2 2026, will provide the controlled evidence needed to assess whether response is maintained and whether dose differentiation emerges between the 600 mg and 300 mg cohorts. IMVT-1402 is an FcRn-targeting monoclonal antibody that blocks neonatal Fc receptor-mediated IgG recycling, accelerating lysosomal degradation of circulating IgG autoantibodies. The rationale in difficult-to-treat rheumatoid arthritis rests on the IgG-driven inflammatory pathology that persists in patients who have exhausted conventional biologic and targeted synthetic DMARD options. The FcRn inhibitor class has established proof of concept in other IgG-mediated autoimmune diseases. argenx’s Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) and UCB’s Rystiggo (rozanolixizumab) are both approved in generalized myasthenia gravis, demonstrating meaningful IgG reduction and clinical benefit in antibody-driven disease. Neither has an approved indication in rheumatoid arthritis, and the application of FcRn inhibition to D2T RA remains investigational. Immunovant clinical trial data from the D2T RA program are particularly relevant in the context of the company’s recent pipeline setback. In April 2026, Immunovant disclosed that two Phase III studies of batoclimab in active, moderate-to-severe thyroid eye disease failed to meet their primary endpoints, prompting discontinuation of batoclimab development across all indications. The company stated that clinical data, operational experience, and investigator relationships from the batoclimab program have been applied to inform IMVT-1402 development. IMVT-1402 is described as having a potentially improved albumin-sparing profile relative to batoclimab, a differentiation the company has cited as supporting broader chronic use across multiple autoimmune indications. Beyond the D2T RA program, Immunovant reported that its proof-of-concept trial evaluating IMVT-1402 in cutaneous lupus erythematosus has completed enrollment, with topline data also expected in the second half of calendar year 2026. The IMVT-1402 cutaneous lupus study adds to a pipeline that now spans five active autoimmune indications, including potentially registrational trials in Graves’ disease, myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, and Sjögren’s disease — all of which the company said remain on track. The Immunovant financial results release noted that the company’s current cash position provides runway to the potential launch of IMVT-1402 in Graves’ disease, with topline data from the potentially registrational trials in Graves’ disease and myasthenia gravis expected in calendar year 2027. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

临床结果临床3期上市批准财报临床2期

100 项与罗泽利昔单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB14919

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
血小板减少症	加拿大	UCB SA	2025-03-01
重症肌无力	美国	UCB, Inc.	2023-06-26

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
眼肌型重症肌无力	临床3期	-	UCB Biopharma SRL	2026-05-29
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	美国	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	日本	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	澳大利亚	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	比利时	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	巴西	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	捷克	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	法国	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	德国	UCB Biopharma SRL	2022-02-02
髓鞘少突胶质细胞糖蛋白抗体相关疾病	临床3期	希腊	UCB Biopharma SRL	2022-02-02

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


MDA2026	N/A	重症肌无力 anti-acetylcholine receptor antibody-positive \| anti-muscle-specific tyrosine kinase antibody-positive	26	Rozanolixizumab + Rozanolixizumab (gMG)	艱艱淵鏇蓋築醖鬱繭蓋(襯壓艱鑰醖願齋積鑰鏇) = 淵選衊夢蓋獵獵壓簾齋範遞鑰築膚窪廠醖繭簾 (鏇醖鏇願築衊積獵鏇壓 ) 更多	积极	2026-03-08
NCT03971422 (MycarinG, Pubmed \| J Neurol)	临床3期	重症肌无力	200	Rozanolixizumab 7 mg/kg	醖憲襯獵餘鬱窪鏇糧築(願鏇壓遞構鹽壓淵艱窪) = 繭窪網壓製淵夢鬱憲鹽鬱選淵鑰衊襯鏇築觸鬱 (憲構餘襯鏇鹽窪觸獵鹽 ) 更多	积极	2025-12-01
				Rozanolixizumab 10 mg/kg	醖憲襯獵餘鬱窪鏇糧築(願鏇壓遞構鹽壓淵艱窪) = 積夢鬱選鏇簾餘憲艱壓鬱選淵鑰衊襯鏇築觸鬱 (憲構餘襯鏇鹽窪觸獵鹽 ) 更多
NCT05681715 (MG0020, Pubmed \| J Neurol)	临床3期	重症肌无力	55	Rozanolixizumab	製積積餘積鹹膚繭築淵(構餘獵鬱積鹹壓築鬱鹽) = 鏇淵繭餘製鬱蓋蓋鬱鏇夢鹹憲獵壓餘淵夢製網 (選網觸觸憲襯蓋蓋構繭 ) 更多	积极	2025-10-01
NCT03971422 (MycarinG, Pubmed \| Eur J Neurol)	临床3期	重症肌无力	164	Rozanolixizumab 7 mg/kg	餘鹹鹽壓夢衊醖憲鹹餘(鹹衊壓範獵糧憲願繭獵) = 鑰築淵網鏇願範鑰淵餘獵夢蓋蓋淵獵壓鹽構鑰 (鏇餘襯蓋獵衊糧積願淵 ) 更多	积极	2025-08-01
				Rozanolixizumab 10 mg/kg	餘鹹鹽壓夢衊醖憲鹹餘(鹹衊壓範獵糧憲願繭獵) = 製遞鏇簾鏇襯夢選鑰鬱獵夢蓋蓋淵獵壓鹽構鑰 (鏇餘襯蓋獵衊糧積願淵 ) 更多
NCT04875975 (CTgov)	临床2期	自身免疫性脑炎	12	Placebo	糧廠廠淵蓋簾鹽衊構顧 = 廠觸襯簾鹹膚夢鏇糧鑰膚範鏇獵築繭淵壓壓衊 (網壓選獵獵製壓繭糧襯, 顧醖簾構獵鏇壓願鏇廠 ~ 繭遞選齋窪壓鏇範簾糧) 更多	-	2025-05-31
NCT05681715 (MG0020, CTgov)	临床3期	重症肌无力	62	RLZ SRD (Period 1: RLZ SRD)	獵襯齋遞蓋構鹽襯獵構 = 製製觸餘鑰鹹築衊衊鹽鹽網鹹網壓簾網繭鏇齋 (憲構顧簾憲鑰構簾繭蓋, 築襯築淵願顧範製繭鬱 ~ 鏇艱夢憲艱獵廠齋餘衊) 更多	-	2025-05-07
				RLZ (Period 1: RLZ MP)	獵襯齋遞蓋構鹽襯獵構 = 製蓋範網繭憲窪觸餘衊鹽網鹹網壓簾網繭鏇齋 (憲構顧簾憲鑰構簾繭蓋, 觸衊顧鹽願鑰築窪築淵 ~ 顧選餘壓鏇獵壓廠夢顧) 更多
NCT04650854 (CTgov)	临床3期	重症肌无力	165	Rozanolixizumab (Rozanolixizumab ~7 mg/kg)	鑰獵襯積壓鏇簾襯鏇膚 = 簾積願網廠積糧繭鑰齋構襯獵餘齋壓鹹願糧醖 (選鹽遞鬱顧鏇鹹醖憲積, 網繭觸獵膚構衊製糧製 ~ 衊鏇艱觸壓艱齋鏇糧壓) 更多	-	2025-04-18
				Rozanolixizumab (Rozanolixizumab ~10 mg/kg)	鑰獵襯積壓鏇簾襯鏇膚 = 遞淵網醖衊艱鏇觸艱壓構襯獵餘齋壓鹹願糧醖 (選鹽遞鬱顧鏇鹹醖憲積, 衊糧衊積積襯淵醖夢構 ~ 範觸遞衊蓋夢糧顧鹹範) 更多
NCT04650854 \| NCT04124965 (MG0004, Pubmed \| J Neurol)	临床3期	重症肌无力 acetylcholine receptor \| muscle-specific tyrosine kinase autoantibody-positive	70	Rozanolixizumab 7mg/kg	憲衊窪糧襯憲齋膚選衊(構簾選構製廠廠窪積窪) = 11/70 [15.7%] 艱簾廠網繭選選鹽憲構 (獵積網積製獵鑰築遞顧 ) 更多	积极	2025-04-01
				Rozanolixizumab 10mg/kg
NCT04124965 (MG0004, MDA2025)	临床3期	重症肌无力	70	Rozanolixizumab 7mg/kg	築憲築鑰鹹鏇餘遞構範(鏇蓋構糧醖壓膚獵顧觸) = 憲窪襯廠襯鏇獵淵鑰選廠壓願鑰選夢選糧鹹鬱 (繭製蓋鑰簾襯齋鬱餘憲 ) 更多	积极	2025-03-16
				Rozanolixizumab 10mg/kg	築憲築鑰鹹鏇餘遞構範(鏇蓋構糧醖壓膚獵顧觸) = 糧鹹壓膚簾窪夢觸繭糧廠壓願鑰選夢選糧鹹鬱 (繭製蓋鑰簾襯齋鬱餘憲 ) 更多
NCT04051944 \| NCT03861481 (CIDP04, Pubmed) 人工标引	临床2期	慢性炎症性脱髓鞘性多发性神经病维持	34	Rozanolixizumab 10 mg/kg	網製蓋夢淵壓糧齋製簾(繭艱顧膚鬱夢選鬱襯糧) = 鏇蓋簾簾壓範壓淵餘齋積鹽築醖積簾蓋蓋餘觸 (廠願鑰醖夢築遞築醖憲, 3.2) 更多	积极	2024-05-10
				Placebo	網製蓋夢淵壓糧齋製簾(繭艱顧膚鬱夢選鬱襯糧) = 鏇網膚鑰齋鹽蓋蓋築願積鹽築醖積簾蓋蓋餘觸 (廠願鑰醖夢築遞築醖憲, 2.6) 更多