A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune condition that mainly affects the eyes and spinal cord, causing serious symptoms such as vision loss, paralysis, and severe pain. This trial compares the effectiveness and safety of five medications commonly used to prevent NMOSD relapses: rituximab, ravulizumab, inebilizumab, satralizumab, and eculizumab.
In this study, 160 adults with NMOSD who test positive for a specific antibody (AQP4-IgG) will participate. They will be randomly assigned to receive either rituximab or one of the four other FDA-approved medications. The main goal is to find out which treatment best prevents relapses and has fewer serious side effects. The trial will also measure disability, patient satisfaction, quality of life, and biomarkers that help track disease activity.
Participants will have regular assessments, including medical exams, surveys, and tests for vision, walking ability, and brain function. They will report any side effects or health issues experienced during the study. The trial will last from one to four years for each participant.
This research aims to help patients and doctors make better-informed treatment decisions by providing clear evidence about the best available therapies for NMOSD.

开始日期2025-11-01

申办/合作机构

The Massachusetts General Hospital

[+1]

NCT07024563

/ Not yet recruiting临床3期

A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)

The primary objective of this study is to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population.

开始日期2025-09-26

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT06333652

/ Recruiting临床2期IIT

Clinical Trial on the Use of Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders

The researchers are testing a medication named ravulizumab for the treatment of severe preeclampsia and Hemolysis, Elevated Liver enzymes, Low Platelets (HELLP) syndrome.

开始日期2025-09-01

申办/合作机构

Mayo Clinic

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100 项与瑞利珠单抗相关的转化医学

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100 项与瑞利珠单抗相关的专利（医药）

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284

项与瑞利珠单抗相关的文献（医药）

2025-06-01·JOURNAL OF NEUROLOGY

Ravulizumab for generalized Myasthenia Gravis: a multicenter real-life experience

Article

作者: Marini, Sofia ; Rini, Nicasio ; Habetswallner, Francesco ; Ricciardi, Dario ; Falso, Silvia ; Leonardi, Luca ; Marando, Demetrio ; Fionda, Laura ; Pennisi, Elena Maria ; Di Stefano, Vincenzo ; Tufano, Laura ; Maestri, Michelangelo ; Guida, Melania ; Di Martino, Giuseppe ; Iorio, Raffaele ; Vinciguerra, Claudia ; Antonini, Giovanni ; Morino, Stefania ; Garibaldi, Matteo ; Rossini, Elena

Abstract:

Introduction:

Ravulizumab, a monoclonal antibody targeting C5, was recently approved for the treatment of anti-AChR positive generalized myasthenia gravis (gMG) patients. The objective of this study is to present the Italian multicenter real-world experience evaluating the safety and efficacy of ravulizumab in gMG within the context of the Expanded Early Access Program (EAP).

Methods:

We conducted a retrospective study in 7 gMG referral centres in Italy. Demographic and clinical characteristics were recorded at baseline and during follow-up through clinical scale changes including Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC). Frequency of minimal symptom expression (MSE) and changes in concomitant medications were also evaluated.

Results:

Twenty-four gMG patients (10/24 females) aged between 24 and 82 years (Median 60.5, IQR 52.5–67.5), were included. Fifteen patients had undergone thymectomy, and 14 had a thymoma. Median follow-up duration was 26 weeks (range 10–74, IQR 26–42). MG-ADL and QMG scores showed a significant decrease with respect to baseline (p < 0.001). MSE was achieved by 37.5% patients at the last available follow-up. Tapering of prednisone daily dosage was possible in 76% of patients. Thymoma was significantly associated with QMG score reduction and the frequency of QMG responders at week 2 (p = 0.03).Three patients discontinued treatment. One patient experienced a myasthenic exacerbation and needed rescue therapy. Infectious adverse events were reported in 5/24 patients, and a Stevens-Johnson syndrome in one patient.

Conclusions:

Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.

2025-06-01·JOURNAL DE MYCOLOGIE MEDICALE

Disseminated infection with Candida dubliniensis after ravulizumab and treatment with rezafungin – a case report

Article

作者: Omic, Haris ; Aigner, Christof ; Vossen, Matthias G ; Muraközy, Gabriella ; Lötsch, Felix ; Scharitzer, Martina ; Eder, Michael ; Doberer, Konstantin ; Willinger, Birgit

2025-06-01·Lancet Haematology

Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors

Article

作者: Griffin, Morag ; Mauad, Vitor A Q ; Yang, Chen ; Di Bona, Eros ; Langemeijer, Saskia M C ; Uchiyama, Michihiro ; Maitra, Samopriyo ; Panse, Jens ; Röth, Alexander ; Pullarkat, Vinod ; Maciejewski, Jaroslaw P ; Barcellini, Wilma ; Marano, Luana ; Ferber, Philippe ; Gandhi, Shreyans ; Risitano, Antonio M ; Han, Bing ; Höchsmann, Britta ; Kitawaki, Toshio ; Solar-Yohay, Susan ; Lee, Lily Wong Lee ; Kutlar, Abdullah ; Ueda, Yasutaka ; Mahajan, Navin ; Tavitian, Suzanne ; Chew, Lee Ping ; Zhang, Li ; Jang, Jun Ho ; Frieri, Camilla ; Schafhausen, Philippe ; Li, Shujie ; Dahlke, Marion ; Kelly, Richard J ; Beggiato, Eloise ; de Latour, Régis Peffault ; Terriou, Louis ; Kulasekararaj, Austin G ; Schubert, Jörg ; Trikha, Roochi ; Fu, Rong ; Huang, Wei-Han ; Liu, Hui ; Gaya, Anna ; de Castro, Carlos M ; Yap, Eng-Soo ; Thorburn, Christine ; Verles, Aurelie ; de Fontbrune, Flore Sicre ; Wang, Zhixin ; Scheinberg, Phillip ; Schrezenmeier, Hubert ; Kerloëguen, Cécile ; Lawniczek, Tomasz ; Kumar, Rakesh ; Notaro, Rosario ; Alashkar, Ferras

BACKGROUND:

The factor B inhibitor iptacopan improved 24-week outcomes in adult patients with paroxysmal nocturnal haemoglobinuria in the phase 3 APPLY-PNH and APPOINT-PNH trials; the trial extension periods assessed clinical activity and safety up to 48 weeks. Here, we report the final 48-week data from APPLY-PNH and APPOINT-PNH.

METHODS:

In both APPLY-PNH and APPOINT-PNH trials, patients were aged 18 years or older, with paroxysmal nocturnal haemoglobinuria (red and white blood cell population sizes ≥10%) and without laboratory evidence of bone marrow failure. In APPLY-PNH (an open-label, randomised, phase 3 trial conducted in 39 centres [38 hospitals, one outpatient research clinic] from 12 countries or regions), patients with haemoglobin concentration lower than 10 g/dL on anti-C5 treatment (stable eculizumab or ravulizumab regimen for ≥6 months) were randomly assigned (8:5) via interactive response technology to either receive oral iptacopan 200 mg twice daily (iptacopan group) or to continue their individual intravenous eculizumab or ravulizumab regimen for 24 weeks (anti-C5 group). Randomisation was stratified by type of anti-C5 and receipt of red blood cell (RBC) transfusions in the preceding 6 months. In APPOINT-PNH (an open-label, single-arm, phase 3 trial conducted in 12 hospitals from eight countries), complement inhibitor-naive patients with paroxysmal nocturnal haemoglobinuria and with haemoglobin concentration lower than 10 g/dL and lactate dehydrogenase (LDH) concentration higher than 1·5 times the upper limit of normal received iptacopan 200 mg twice daily for 24 weeks. Both trials had 24-week extension periods in which all patients received iptacopan monotherapy. Primary endpoints were the proportion of patients with an increase from baseline in haemoglobin concentration of 2 g/dL or higher (APPLY-PNH and APPOINT-PNH) and haemoglobin concentration 12 g/dL or higher (APPLY-PNH) between weeks 18 and 24, all in the absence of RBC transfusions between weeks 2 and 24; results for these primary endpoints have been reported previously. We report final activity and safety data at the completion of both trials (week 48). Prespecified endpoints at week 48 included percentage of patients with a haemoglobin increase from baseline of 2 g/dL or higher or haemoglobin 12 g/dL or higher (including post-transfusion data). Efficacy data were analysed per the intention-to-treat principle, and safety was analysed according to the treatment that patients received. APPLY-PNH and APPOINT-PNH are registered with ClinicalTrials.gov, NCT04558918 and NCT04820530, respectively.

FINDINGS:

In APPLY-PNH, between Jan 25, 2021, and April 8, 2022, 62 patients (43 [69%] female, 19 [31%] male; 48 [77%] White, 12 [19%] Asian, two [3%] Black) were randomly assigned to the iptacopan group and 35 patients (24 [69%] female, 11 [31%] male; 26 [74%] White, seven [20%] Asian, two [6%] Black) to the anti-C5 group; 61 (98%) and 34 (97%), respectively, entered the extension period. At trial completion (March 6, 2023), the median duration of iptacopan treatment was 337 days (IQR 168-338). In APPOINT-PNH, 40 patients were enrolled between July 19, 2021, and May 17, 2022, and received iptacopan (17 [43%] female, 23 [58%] male; 12 [30%] White, 27 [68%] Asian, one [3%] Black); all entered the extension period. At trial completion (April 18, 2023), the median duration of iptacopan treatment was 337 days (IQR 337-344). At week 48, irrespective of RBC transfusions, the number of patients who had an increase in haemoglobin concentration of 2 g/dL or higher was 51 (86%) of 59 in the APPLY-PNH iptacopan group, 21 (72%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 38 (97%) of 39 in APPOINT-PNH. The number of patients who had haemoglobin concentration of 12 g/dL or higher at week 48 was 40 (68%) of 59 in the APPLY-PNH iptacopan group, 17 (59%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 31 (79%) of 39 in APPOINT-PNH. There were no treatment discontinuations because of treatment-emergent adverse events or deaths. Across the 48-week trials, clinical breakthrough haemolysis occurred in seven (7%) of 96 iptacopan-treated patients in APPLY-PNH (including both groups) and two (5%) of 40 in APPOINT-PNH, but it was generally mild or moderate with no iptacopan discontinuation. Three major adverse vascular events occurred in APPLY-PNH by trial completion; all were considered unrelated to iptacopan. The most common treatment-emergent adverse event was COVID-19 in APPLY-PNH (iptacopan: 18/62 patients [29%]; anti-C5-to-iptacopan: 8/34 [24%]) and headache in APPOINT-PNH (12/40 [30%]). Severe and serious treatment-emergent adverse events were experienced by six (10%) and nine (15%) of 62 patients in the APPLY-PNH iptacopan group, respectively; in APPOINT-PNH, these were experienced by four (10%) and eight (20%) of 40 patients, respectively. The most common serious treatment-emergent adverse event was COVID-19, occurring in one (2%) of 62 patients in the APPLY-PNH iptacopan group and two (5%) of 40 patients in APPOINT-PNH. No severe treatment-emergent adverse events occurred in more than one patient.

INTERPRETATION:

Long-term data indicate durable haemolysis control with iptacopan in paroxysmal nocturnal haemoglobinuria, maintained normal or near-normal haemoglobin, and no new safety concerns. We believe that these data support iptacopan as a potential therapy option, suggesting that we are in a new treatment era for paroxysmal nocturnal haemoglobinuria.

FUNDING:

Novartis.

478

项与瑞利珠单抗相关的新闻（医药）

2025-06-25

·医脉通

补体抑制剂时代下PNH的未尽之需：EHA 2025聚焦需求优化与治疗目标升级

引言2025年第30届欧洲血液学会（EHA）年会已圆满落幕。这场学术盛宴汇聚了全球多国知名学者，共探血液前沿进展，推动血液领域持续发展，为未来诊疗方向注入全新思路。会议期间，PNH专题会紧扣“Elevating treatment goals in paroxysmal nocturnal hemoglobinuria (PNH): meeting patients’ needs in the evolving era of complement inhibition（提升阵发性睡眠性血红蛋白尿症[PNH]治疗目标：在补体抑制治疗演进时代满足患者的需求）”主题，聚焦罕见血液疾病PNH，特邀领域内资深学者深入探讨在补体抑制治疗不断演进的时代背景下，如何提升PNH治疗目标，以切实满足患者的需求，并为PNH的诊疗提供前沿见解。Mapping the terrain：PNH治疗的变革之路PNH是一种罕见、危及生命的获得性造血干细胞克隆性疾病：PNH是由于造血干细胞的PIGA基因突变，导致红细胞表面缺失GPI锚定补体调节蛋白（CD55/CD59），在补体异常激活介导下发生血管内溶血（IVH）1。PNH以溶血、骨髓衰竭（BMF）和血栓形成为主要特征。溶血会导致平滑肌功能障碍，可能表现为吞咽困难、勃起功能障碍或腹痛。持续溶血还可能导致多系统受累，包括肾功能损伤、疲劳、贫血等2。C5补体抑制剂的发展历程和现存局限：C5补体抑制剂可阻止膜攻击复合物（MAC）形成，抑制末端补体介导的IVH，改善患者的临床结局1。2007年，首款C5补体抑制剂获批上市，需每周一次静脉输注；2019年，半衰期更长的C5补体抑制剂获批上市，给药频率为每2周输注1次；2024年，皮下注射的C5补体抑制剂获得欧洲药品管理局（EMA）批准用于PNH患者，给药便捷性提升。尽管接受抗C5治疗，PNH患者仍面临许多未被满足的临床需求和生活质量（QoL）负担。高达82%的患者血红蛋白（Hb）水平未恢复正常3，18~39%的患者仍依赖红细胞（RBC）输注4,5。此外，治疗需要每2周或每8周进行一次静脉输注，每次就医耗时4~5.5小时6，严重影响生活和工作。值得注意的是，高达50%的患者经历血管外溶血（EVH），导致持续性贫血1，75~89%的患者报告出现疲劳7,8。近端补体抑制剂开启PNH治疗新纪元，为提升治疗目标带来机遇:近端补体抑制剂可控制末端补体介导的IVH和C3介导的EVH。通过靶向补体系统的近端通路，为患者带来进一步的血液学获益和QoL改善。目前已有三款近端补体抑制剂获批用于治疗合并溶血性贫血的PNH患者9-11。近端补体抑制剂通过控制IVH和EVH，改善Hb水平，减轻持续性贫血症状，助力患者摆脱输血依赖。此外，还可改善疲劳及健康相关生活质量（HRQoL），包括身体、角色和社会功能等方面1。口服制剂的推出也为患者和医生提供了更多的给药选择。小结补体抑制剂的发展改变了PNH的治疗格局，不同给药途径药物的不断涌现，为医疗专业人员和患者提供了更多选择，使得个性化的治疗决策成为可能，从而更好地满足个体的需求和偏好。尤其是近端补体抑制剂的出现，标志着PNH治疗进入新纪元，实现了从“控制溶血”到“器官保护与生活质量改善”的目标升级。Starting the climb：解析未接受补体抑制剂治疗的伴溶血PNH患者的治疗策略控制IVH是未接受补体抑制剂治疗的PNH患者的关键治疗目标：IVH是未接受补体抑制剂治疗患者中最主要的溶血类型，会引发贫血。未控制的IVH与贫血、输血依赖、HRQoL下降、血红蛋白尿、平滑肌功能障碍、肾脏损伤、肺动脉高压、血栓形成等多种严重并发症密切相关。因此，控制IVH是减轻PNH患者贫血及其他严重并发症的关键。口服伊普可泮单药治疗可快速且持续控制IVH，多数患者Hb水平达到正常或接近正常12：乳酸脱氢酶（LDH）水平从基线开始迅速下降，第48周时LDH均值为305.5 U/L，多数时间维持在1.5倍正常值上限（ULN）以下。第48周，79.5%的患者Hb≥12 g/dL，患者平均Hb水平为13.2 g/dL，97.5%的患者避免输血。在安全性方面，无重大血管事件（MAVEs），仅2例患者发生2起突破性溶血（BTH）事件，无因脑膜炎奈瑟菌、肺炎链球菌或流感嗜血杆菌导致的严重感染。在未接受补体抑制剂治疗的患者中，静脉输注Ravulizumab在控制IVH方面的疗效不劣于依库珠单抗13,14：Ravulizumab单药组、依库珠单抗单药组和依库珠单抗转换为Ravulizumab组的LDH水平随时间变化趋势相近。在开放标签扩展期间，Ravulizumab单药组有超63%的患者实现Hb稳定，75.7%的患者避免输血。在安全性方面，从基线至第6年期间，MAVEs发生率为4.5%，BTH发生率为14.8%，治疗期间出现的严重不良事件（TEAEs）发生率为39.8%。皮下注射Pegcetacoplan在控制IVH方面优于支持治疗15,16：Pegcetacoplan组的LDH水平从基线开始显著下降，支持治疗组无明显变化。第74周时，Pegcetacoplan组中位LDH水平为189 U/L，而支持治疗组为1535 U/L。第74周时，46.4%的患者Hb≥12 g/dL，平均Hb水平为11.6 g/dL。第26周时，91.4%的患者避免输血，而第74周时这一比例仍保持在90.0%。在安全性方面，至第74周时，无血栓事件发生。溶血相关TEAEs发生率在第1-26周为0，第26-74周为12.0%。小结控制IVH是初始治疗PNH患者的关键策略，能够有效降低溶血相关并发症和输血需求。不同补体抑制剂的治疗效果各异，其中，补体B因子抑制剂伊普可泮能够实现全面控制溶血，近80%的患者Hb≥12 g/dL，97.5%的患者成功脱离输血，仅5%的患者发生BTH。Continuing the ascent：提升抗C5治疗后持续性贫血PNH患者的治疗目标C5补体抑制剂治疗后，患者疗效欠佳：接受C5补体抑制剂治疗5年的PNH患者，长期结局存在差异，随着治疗时间的推移，完全缓解等良好反应的比例呈现一定变化趋势17。C5补体抑制剂的疗效受多种因素影响，如骨髓衰竭程度、EVH程度、合并炎症情况、以及Hb水平变化。C5补体抑制剂虽能抑制MAC介导的IVH，但补体旁路途径持续激活会导致C3转化酶生成，介导C3b片段在PNH红细胞表面沉积，进而在肝脏、脾脏被巨噬细胞吞噬，引发EVH。接受C5补体抑制剂治疗的患者中，高达50%的患者存在EVH，是持续性贫血的主要原因1。持续性贫血可导致患者持续依赖RBC输注，进而对患者的HRQoL产生负面影响，还可能引发器官损伤。接受C5补体抑制剂治疗的患者，仍可能报告PNH相关症状，缓解情况不佳。对于抗C5治疗疗效欠佳的患者，可转换近端补体抑制剂：接受抗C5治疗后仍存在持续性贫血的PNH患者，目前有多种近端补体抑制剂可供选择。从C5补体抑制剂转换为近端补体抑制剂时，应考虑患者的临床特征（贫血严重程度、合并症、既往补体抑制剂治疗的反应、血栓事件病史、支持治疗史、BMF程度、妊娠情况、基因特征）和生活方式（治疗所需的交通时间和便利性、偏好的给药途径等）。近端补体抑制剂单药或联合C5补体抑制剂，可为C5疗效欠佳的患者带来希望：口服伊普可泮单药治疗可实现全面的溶血控制，在减少EVH和提升Hb水平方面均优于C5补体抑制剂12,18。伊普可泮可摆脱输血依赖，并将疲劳改善至正常水平，减轻患者负担。伊普可泮治疗48周后，BTH发生率为7.0%。仅3例MAVEs，且均与伊普可泮无关。无脑膜炎奈瑟菌、肺炎链球菌或流感嗜血杆菌导致的严重感染。与抗C5治疗联合安慰剂相比，Danicopan联合抗C5治疗在减少EVH和提升Hb水平方面更具优势，避免输血的患者比例更高，疲劳改善更显著19,20。与C5补体抑制剂相比，皮下注射Pegcetacoplan可减少EVH，且在提升Hb水平方面更具优势。第16周时，85.0%的患者避免输血，第96周时，慢性疾病治疗功能评估-疲劳（FACIT-F）评分为40.8分21-23。任何补体抑制剂治疗均可能发生BTH，其严重程度决定治疗管理策略：BTH是指在初始治疗有反应后，IVH的体征和症状突然再次出现，与LDH水平显著升高和Hb水平降低相关。按发生机制，BTH分为药代动力学相关BTH和药效动力学相关BTH。前者通常是由于补体抑制剂的血药浓度低于有效浓度；后者通常是由于感染或炎症等补体扩增条件（CAC）引发强烈的补体激活并突破补体抑制剂的阻断作用。临床中常依据BTH的严重程度进行管理。对于轻度BTH，无需治疗（监测Hb/LDH水平及BTH症状），或治疗潜在的CAC。对于中至重度BTH，则需针对发病机制采取应对策略，并按需处理BTH并发症，抗血栓治疗等。小结对于接受抗C5治疗后仍存在持续性贫血的PNH患者，全面控制溶血是解决问题的关键。近端补体抑制剂已被批准用于这部分患者的治疗，其中，口服伊普可泮单药治疗能够有效实现Hb正常化、摆脱输血依赖并改善疲劳症状，且BTH发生率较低，显示出良好的临床应用前景。Reaching new heights：揭示C5补体抑制剂治疗中Hb>10 g/dL患者的未满足需求EVH及其相关并发症是许多接受C5补体抑制剂治疗的PNH患者未被满足的需求：尽管接受C5补体抑制剂治疗，仍有高达82%的PNH患者无法达到正常Hb水平，主要原因是出现了C3介导的EVH、残留IVH以及骨髓衰竭。在接受C5补体抑制剂治疗期间，即使Hb水平为10-12 g/dL或＞12 g/dL的患者，仍可能存在持续EVH。持续EVH可导致PNH患者存在持续性贫血，无法摆脱输血依赖，这会对患者的HRQoL产生负面影响。慢性贫血会给患者（尤其是老年患者）带来显著的健康负担：在女性Hb>10 g/dL但≤12 g/dL，男性Hb>10 g/dL但≤13 g/dL的情况下，患者仍被视为轻至中度贫血。慢性贫血可导致心脏功能改变，是心力衰竭患者病情恶化和死亡的预测因素。尤其在老年患者中，慢性贫血可导致严重并发症。老年贫血是心血管疾病、认知障碍、失眠、情绪障碍、QoL受限、执行功能下降、跌倒和骨折、身体机能降低的危险因素。贫血可导致疲劳，这仍是接受C5补体抑制剂治疗的PNH患者未被满足的需求：贫血可导致疲劳，75~89%的接受C5补体抑制剂治疗的PNH患者存在疲劳，尤其是Hb＜10.5 g/dL的患者，疲劳发生频率更高。疲劳被认为是PNH最令患者虚弱的症状之一，会干扰患者的日常活动和生产力，影响认知功能，对整体健康状况产生显著影响。疲劳的患者较无疲劳症状的患者住院风险更高。小结尽管接受C5补体抑制剂治疗，但由于EVH持续存在，Hb为10-12 g/dL的患者仍存在贫血。对于这部分患者，Hb正常化应作为首要目标，以改善QoL、降低严重并发症风险。总结近端补体抑制剂的获批，标志着PNH治疗进入新时代，自此可根据个体需求定制个性化治疗方案。控制IVH是未接受过补体抑制剂治疗患者的关键需求，抗C5治疗虽然可以减少IVH，但常伴随EVH的发生，因此降低持续性贫血成为关键目标。口服Danicopan（联合C5补体抑制剂）和皮下注射Pegcetacoplan单药治疗，可减少C5补体抑制剂经治患者的IVH和EVH，改善血液学结局、减轻患者负担；Pegcetacoplan用于未接受过补体抑制剂治疗的患者时，也有类似效果。口服伊普可泮单药治疗，可全面控制C5补体抑制剂经治及未接受过补体抑制剂治疗患者的IVH和EVH，使Hb正常化，摆脱输血依赖，显著改善疲劳症状。对于接受C5补体抑制剂治疗后，Hb为10-12 g/dL且仍存在贫血的患者，Hb正常化应作为其首要目标，以改善QoL、降低严重并发症风险。参考文献：1.Risitano AM, et al. Front Immunol. 2019 Jun 14;10:1157.2.Cançado RD, et al. Hematol Transfus Cell Ther. 2021 Jul-Sep;43(3):341-348.3.Fishman J et al. Hematol Rep 2023;15:266-82.4.Debureaux PE et al. Bone Marrow Transplant 2021;56:2600-2.5.Schrezenmeier H et al. Ther Adv Hematol 2020;111:1-14.6.Levy AR et al. Blood 2019;134:4803.7.Dingli D et al. Ann Hematol 2022;101:251-63.8.Young NS et al. Semin Hematol 2009;46:S1-16.9.Sobi. Aspaveli® SmPC, 2024. Available at: https://www.ema.europa.eu/en/documents/product-information/aspaveli-epar-product-information_en.pdf (accessed June 2025).10.Alexion Europe SAS. VOYDEYA™ SmPC, 2025. Available at: https://www.ema.europa.eu/en/documents/product-information/voydeya-epar-product-information_en.pdf (accessed June 2025).11.Novartis. FABHALTA® SmPC, 2025. Available at: https://www.ema.europa.eu/en/documents/product-information/fabhalta-epar-product-information_en.pdf (accessed June 2025).12.Risitano AM et al. Lancet Haematol 2025;12:e414-30.13.Lee JW et al. Blood 2019;133:530-9.14.Kulasekararaj A et al. Ann Hematol 2025;104:81-94.15.Wong RSM et al. Blood Adv 2023;7:2468-78.16.Patriquin C et al. Adv Ther 2024;41:2050-69.17.Versmold K et al. Eur J Haematol 2023;111:84-95.18.Peffault de Latour R et al. N Engl J Med 2024;390:994-1008.19.Kulasekararaj A, et al. Blood. 2025 Feb 20;145(8):811-822.20.Lee JW et al. Lancet Haematol 2023;10:e955-65.21.Patriquin C et al. Adv Ther 2024;41:2050-69.22.Hillmen P et al. N Engl J Med 2021;384:1028-37.23.Peffault de Latour R et al. Lancet Haematol 2022;9:e648-59.“本文仅供医疗卫生专业人士参考”审批码FAP0049052-104070，有效期为2025-06-24至2026-06-24，资料过期，视同作废撰写：Isa审校：Vera排版：Zelda执行：Atai本平台旨在为医疗卫生专业人士传递更多医学信息。本平台发布的内容，不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议。如该等信息被用于了解医学信息以外的目的，本平台不承担相关责任。本平台对发布的内容，并不代表同意其描述和观点。若涉及版权问题，烦请权利人与我们联系，我们将尽快处理。

临床研究上市批准

2025-06-22

·信狐药迅

康希诺-国产13价肺炎球菌多糖结合疫苗上市| 新获批(6.16-6.22）

每周药品注册获批数据，分门别类呈现，一目了然。(6.16-6.22）新药上市申请药品名称企业注册分类受理号13价肺炎球菌多糖结合疫苗(CRM197,TT载体)康希诺生物股份公司2.2CXSS2400020新药临床申请药品名称企业注册分类受理号HRS-8829注射用浓溶液北京盛迪医药有限公司1CXHL2500379艾本那肽注射液常山凯捷健生物药物研发(河北)有限公司1CXHL2500378注射用TFX05-01浙江扬厉医药技术有限公司1CXHL2500373TQH3906胶囊正大天晴药业集团南京顺欣制药有限公司1CXHL2500371TQH3906胶囊正大天晴药业集团南京顺欣制药有限公司1CXHL2500370DZD6008片迪哲(江苏)医药股份有限公司1CXHL2500376DZD6008片迪哲(江苏)医药股份有限公司1CXHL2500375DAT-1604片北京丹擎医药科技有限公司1CXHL2500364DAT-1604片北京丹擎医药科技有限公司1CXHL2500363WEYZ-1乳膏南京韦尔优众医药有限公司1CXHL2500365177Lu-NRT6020注射液成都纽瑞特医疗科技股份有限公司1CXHL250035968Ga-NRT6020注射液成都纽瑞特医疗科技股份有限公司1CXHL2500358VCT220片成都闻泰医药科技有限公司1CXHL2500355VCT220片成都闻泰医药科技有限公司1CXHL2500354VCT220片成都闻泰医药科技有限公司1CXHL2500353AOD113408注射液成都奥达生物科技有限公司1CXHL2500339EDP167注射液亿腾医药(珠海)有限公司1CXHL2500336JKN2301干混悬剂健康元药业集团股份有限公司1CXHL2500327EP-0203XR成都苑东生物制药股份有限公司2.3CXHL2500333舒沃替尼片迪哲(江苏)医药股份有限公司2.4CXHL2500374枸橼酸舒芬太尼注射液江苏恩华药业股份有限公司2.4CXHL2500351枸橼酸舒芬太尼注射液江苏恩华药业股份有限公司2.4CXHL2500350枸橼酸舒芬太尼注射液江苏恩华药业股份有限公司2.4CXHL2500349枸橼酸舒芬太尼注射液江苏恩华药业股份有限公司2.4CXHL2500348ZHCX002九华华源药业股份有限公司2.4CXHL2500313流感病毒裂解疫苗(BK-01佐剂)长春百克生物科技股份公司3.2CXSL2500239SCTB39-1注射液神州细胞工程有限公司1CXSL2500364AI-081注射液南京昂科免疫生物医药有限公司1CXSL2500304PM1300注射用冻干制剂珠海普米斯生物科技有限公司1CXSL2500290注射用HY0001a四川汇宇海玥医药科技有限公司1CXSL2500294LNF2102注射液山东新时代药业有限公司1CXSL2500287注射用QLS32015齐鲁制药有限公司1CXSL2500285注射用LY4101174礼来苏州制药有限公司1CXSL2500272注射用ZG006苏州泽璟生物制药股份有限公司1CXSL2500279注射用ZG005苏州泽璟生物制药股份有限公司1CXSL2500278CM512注射液康诺亚生物医药科技(成都)有限公司1CXSL2500275ZHB118舌下片江苏众红生物工程创药研究院有限公司1CXSL2500264ZHB118舌下片江苏众红生物工程创药研究院有限公司1CXSL2500263IMC-003宜明凯尔生物医药技术(上海)有限公司1CXSL2500261HD006细胞华道(上海)生物医药有限公司1CXSL2500251QL2109注射液齐鲁制药有限公司2.2CXSL2500284仿制药申请药品名称企业注册分类受理号盐酸苯海拉明注射液华夏生生药业(北京)有限公司3CYHS2501576复方电解质注射液(V)河北天成药业股份有限公司3CYHS2400745法莫替丁注射液海南全星制药有限公司3CYHS2400674氯化钙注射液华夏生生药业(北京)有限公司3CYHS2400602尼麦角林片浙江赛默制药有限公司3CYHS2400518己酮可可碱注射液辰欣药业股份有限公司3CYHS2400498法莫替丁注射液东阳祥昇医药科技有限公司3CYHS2400342美洛昔康注射液石药集团中诺药业(石家庄)有限公司3CYHS2400303阿奇霉素颗粒重庆莱美药业股份有限公司3CYHS2400199盐酸西替利嗪口服溶液福建海西新药创制股份有限公司3CYHS2303555注射用盐酸罗沙替丁醋酸酯山东豪瑞恩制药有限公司3CYHS2303400醋酸葡萄糖维持液仁合益康集团有限公司3CYHS2303177醋酸葡萄糖维持液仁合益康集团有限公司3CYHS2303176布洛芬注射液陕西丽彩药业有限公司3CYHS2303063布洛芬注射液陕西丽彩药业有限公司3CYHS2303062重酒石酸间羟胺注射液弘和制药有限公司3CYHS2302971硫辛酸片浙江恒研医药科技有限公司3CYHS2302913醋酸钙片青岛百洋制药有限公司3CYHS2302887地氯雷他定口服溶液湖南嘉恒制药有限公司3CYHS2302849己酮可可碱注射液浙江北生药业汉生制药有限公司3CYHS2302843异烟肼注射液海南秋实医药科技有限公司3CYHS2302710氨溴特罗口服溶液南京易腾药物研究院有限公司3CYHS2302510磷酸奥司他韦干混悬剂恒昌(广州)新药研究有限公司3CYHS2302257地塞米松磷酸钠注射液汇禹远和(海南)药业有限公司3CYHS2300998枸橼酸铋钾片浙江京新药业股份有限公司4CYHS2402389非诺贝特酸胆碱缓释胶囊湖南九典制药股份有限公司4CYHS2401389阿司匹林肠溶片浙江易泽达医药科技有限公司4CYHS2401356贝前列素钠片山东百诺医药股份有限公司4CYHS2401345达肝素钠注射液辰欣药业股份有限公司4CYHS2401232达肝素钠注射液辰欣药业股份有限公司4CYHS2401231盐酸达泊西汀片浙江北生药业汉生制药有限公司4CYHS2400915盐酸达泊西汀片昆山培力药品有限公司4CYHS2400863复方托吡卡胺滴眼液山东辰欣佛都药业股份有限公司4CYHS2400860盐酸氨溴索口服溶液黑龙江澳利达奈德制药有限公司4CYHS2400828甲氨蝶呤注射液江苏奥赛康药业有限公司4CYHS2400815甲氨蝶呤注射液江苏奥赛康药业有限公司4CYHS2400814比索洛尔氨氯地平片天大药业(珠海)有限公司4CYHS2400634丙酸氟替卡松乳膏重庆华邦制药有限公司4CYHS2400596夫西地酸乳膏重庆华邦制药有限公司4CYHS2400560注射用唑来膦酸浓溶液广州市联瑞制药有限公司4CYHS2400505盐酸达泊西汀片郑州德迈药业有限公司4CYHS2400522注射用硫酸艾沙康唑山西德元堂药业有限公司4CYHS2400392盐酸氨溴索口服溶液湖北凯纳药业有限公司4CYHS2400216盐酸氨溴索口服溶液湖北凯纳药业有限公司4CYHS2400215磷霉素氨丁三醇颗粒苏州二叶制药有限公司4CYHS2400128西甲硅油乳剂成都倍特得诺药业有限公司4CYHS2400109布洛芬混悬液河南省尤里卡生物科技有限公司4CYHS2400070布洛芬混悬液河南省尤里卡生物科技有限公司4CYHS2400069瑞巴派特片东阳祥昇医药科技有限公司4CYHS2400078蒙脱石散郑州泰丰制药有限公司4CYHS2303493铝碳酸镁咀嚼片太康海恩药业有限公司4CYHS2303433他氟前列素滴眼液江苏安必生制药有限公司4CYHS2303258吸入用布地奈德混悬液江苏西普拉制药有限公司4CYHS2303217富马酸伏诺拉生片宁波科尔康美诺华药业有限公司4CYHS2303018富马酸伏诺拉生片宁波科尔康美诺华药业有限公司4CYHS2303017布南色林片河北龙海药业有限公司4CYHS2302617左卡尼汀口服溶液吉林省辉南长龙生化药业股份有限公司4CYHS2301985唑来膦酸注射液吉林四长制药有限公司4CYHS2300987盐酸乌拉地尔注射液北京民康百草医药科技有限公司4CYHS2300535四价流感病毒裂解疫苗复星雅立峰(大连)生物制药有限公司3.3CXSS2200083利妥昔单抗注射液山东新时代药业有限公司3.3CXSS2400015达普司他片齐鲁制药(海南)有限公司3CYHL2500085达普司他片齐鲁制药(海南)有限公司3CYHL2500084达普司他片齐鲁制药(海南)有限公司3CYHL2500083达普司他片齐鲁制药(海南)有限公司3CYHL2500082镥[177Lu]氧奥曲肽注射液迈得诺医疗科技集团有限公司3CYHL2500076盐酸达克罗宁局部溶液剂深圳珐玛易药品科技有限公司3CYHL2500073沙美特罗替卡松吸入粉雾剂杭州知兴制药有限公司4CYHL2500068吸附破伤风疫苗北京智飞绿竹生物制药有限公司3.3CXSL2500250流感病毒裂解疫苗陕西昆秦生物科技有限公司3.3CXSL2500243人生长激素注射液亿帆医药(上海)有限公司3.3CXSL2500289进口申请药品名称企业注册分类受理号盐酸达利雷生片Idorsia Pharmaceuticals Deutschland GmbH5.1JXHS2400050盐酸达利雷生片Idorsia Pharmaceuticals Deutschland GmbH5.1JXHS2400049左甲状腺素钠片Aristo Pharma GmbH5.2JYHS2400034左甲状腺素钠片Aristo Pharma GmbH5.2JYHS2400033重酒石酸间羟胺注射液Wockhardt Bio Pty Ltd.5.2JYHS2400006注射用醋酸卡泊芬净Gland Pharma Limited5.2JYHS2300034骨化三醇软胶囊Strides Pharma Global Pte Limited5.2JYHS2200010国;JYHS2200010骨化三醇软胶囊Strides Pharma Global Pte Limited5.2JYHS2200009国;JYHS2200009Casdatifan 片Arcus Biosciences, Inc.1JXHL2500076奥拉帕利片AstraZeneca AB2.4JXHL2500083奥拉帕利片AstraZeneca AB2.4JXHL2500082ABBV-324注射用粉末AbbVie Inc.1JXSL2500055MEDI5752AstraZeneca AB1JXSL2500056Isatuximab注射液(皮下注射)Sanofi-Aventis Recherche & Développement2.1JXSL2500053Ravulizumab注射液Alexion Pharmaceuticals, Inc.2.2JXSL2500052Ravulizumab注射液Alexion Pharmaceuticals, Inc.2.2JXSL2500051中药相关申请无注：橙色字体部分结论为不批准或收到通知件；

疫苗申请上市上市批准

2025-06-21

·astrazeneca.com

Alexion data presented at EAN 2025 highlights its leadership and commitment to improving patient outcomes in rare neurological diseases

Alexion, AstraZeneca Rare Disease, will present seven abstracts, including four oral presentations, from its leading rare neurology portfolio at the European Academy of Neurology (EAN) Annual Congress in Helsinki, Finland, 21 to 24 June 2025. Presentations will discuss real-world evidence related to the use of Ultomiris (ravulizumab) as a potential steroid-sparing therapy and build on the demonstrated safety and efficacy profiles of Ultomiris and Soliris (eculizumab) in adult patients with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalised myasthenia gravis (gMG). Christophe Hotermans, Head of Global Medical Affairs, Alexion, said: “At this year’s EAN, Alexion’s data will build upon our leadership in rare neurology. This includes real-world evidence which underscores the clinical benefit of sustained treatment with Ultomiris in adults with AChR-Ab+ gMG. Alexion data will reinforce the potential for Ultomiris to help achieve key treatment goals for individuals living with gMG, including symptom management and reduced steroid burden, and provide meaningful insights to help improve patient care.” New analyses further support steroid-sparing benefit of Ultomiris in gMG Data will be presented in two separate oral presentations evaluating the real-world impact of rapid and sustained C5 inhibition with ravulizumabon reducing the burden of oral corticosteroid (OCS) use in patients with AChR-Ab+ gMG. Clinical practice outcomes data from the global MG SPOTLIGHT registry will show that a reduction in concomitant immunosuppressive therapy and OCS burden was observed in adults with AChR-Ab+ gMG, following ravulizumabtreatment initiation, supporting its potential steroid-sparing role. Data from 44 patients analysed will show that 10/33 (30.3%) of patients receiving one or more concomitant immunosuppressive therapies at initiation discontinued at least one therapy following treatment with ravulizumab. In addition, after six months, the number of patients receiving ≤5 and ≤10 mg/day OCS increased from 11/26 (42.3%) and 16/26 (61.5%), respectively, to 16/26 (61.5%) and 20/26 (76.9%). Separately, observations from a retrospective analysis of medical records from adults with AChR-Ab+ gMG who initiated treatment with either ravulizumabor efgartigimod will show a potential trend toward greater reduction in OCS use and fewer event-related hospitalisations following treatment with ravulizumab. Data will show that among patients taking OCS at initiation, 17/19 (89.5%) ravulizumabtreated patients and 33/46 (71.7%) efgartigimod treated patients reduced/discontinued their OCS dose post-initiation. In addition, mean exacerbation-related hospitalisations per patient per month decreased from 0.17 to 0 among ravulizumabtreated patients and increased from 0.19 to 0.25 among efgartigimod treated patients. Real-world data reflects improved aspects of daily function in gMG patients treated with Ultomiris or Soliris An oral presentation of data from the global MG SPOTLIGHT registry will show that in adults with AChR-Ab+ gMG, treatment with ravulizumabor eculizumab resulted in statistically significant improvements in different aspects of the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores after treatment began (P<0.05). Patients on ravulizumabor eculizumab experienced complete or partial reductions in symptoms affecting vision (60.7% and 63.3%), speech (58.4% and 59.2%), breathing (33.7% and 26.5%) and mobility (51.7% and 49.0%). Similar improvements were seen when switching from eculizumab toravulizumab(67.5% (vision), 67.5% (speech), 45.0% (breathing) and 65.0% (mobility)). Advancing innovation in gMG and awareness of the impact of misdiagnosis in NMOSD An oral presentation will share results from Ad Scientiam’s decentralised research study, funded by Alexion, to evaluate the use of the app-based tool, ME&MG openTM for remote, daily tracking of gMG symptoms in adults with AChR-Ab+ gMG. These data will show that most data met quality criteria and that more than 65% of patients remained adherent after one year, highlighting the feasibility of digital biomarker collection for a broad gMG population. A poster presentation will share baseline characteristics of participants enrolled in the PREVAIL Phase III trial evaluating the safety and efficacy of gefurulimab, an investigational novel, dual-binding nanobody, optimised for weekly subcutaneous administration, in adult patients with AChR-Ab+ gMG. A virtual poster will provide an overview of the ongoing Phase III global study evaluating the safety and efficacy of gefurulimab for paediatric patients with AChR-Ab+ gMG. Another virtual poster will showcase findings from a European, cross-sectional survey of physicians and anti-aquaporin-4 (AQP4) antibody positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) patients. Results will demonstrate that patients initially misdiagnosed compared to those correctly diagnosed with AQP4-Ab+ NMOSD were more likely to experience physical disability (37.6% vs 24.5%, p=0.027), require caregiver involvement (61.0% vs 43.6%, p=0.006) and need help with a higher number of daily activities (mean [SD], 3.4 [2.8] vs 2.1 [1.8], p<0.001). Worsening of quality of life was also reported, emphasising the urgent need for increased understanding to facilitate reduction in time to diagnosis. Lead Author Abstract Title Presentation Details Lead Author gMG Lead Author Scheiner C, et al. Abstract Title Myasthenic Crises, Exacerbations, and Corticosteroid Use in US Patients Receiving Ravulizumab or Efgartigimod Presentation Details Oral Presentation Abstract ID: A-25-13152 21 June 2025 14:10 – 14:15 EEST Lead Author Saccà F, et al. Abstract Title Assessing Efficacy and Safety of Gefurulimab in Generalised Myasthenia Gravis: Baseline Characteristics From PREVAIL Presentation Details ePoster Presentation Abstract ID: A-25-13135 22 June 2025 13:09 – 13:12 EEST Lead Author Nowak R, et al. Abstract Title Concomitant Immunosuppressive Therapy Use With Ravulizumab: Analysis of a Generalised Myasthenia Gravis Global Registry Presentation Details Oral Presentation Abstract ID: A-25-13153 23 June 2025 14:10 – 14:15 EEST Lead Author Barnett-Tapia C, et al. Abstract Title Overcoming Challenges in Digital Biomarker Collection for Myasthenia Gravis: Insights from the ME&MGopen Decentralised Study* Presentation Details Oral Presentation Abstract ID: A-25-13186 23 June 2025 14:15 – 14:20 EEST Lead Author Juel V, et al. Abstract Title MG-ADL Subdomain Score Changes With Eculizumab or Ravulizumab: A Generalised Myasthenia Gravis Global Registry Analysis Presentation Details Oral Presentation Abstract ID: A-25-13154 23 June 2025 14:25 – 14:30 EEST Lead Author Dy S, et al. Abstract Title Phase 3 Study of Gefurulimab in Paediatric Participants With Generalised Myasthenia Gravis: Trial in Progress Presentation Details Virtual ePoster Presentation Available online on-demand Lead Author NMOSD Lead Author Kleman M, et al. Abstract Title A real-world survey assessing the extent and clinical impact of misdiagnosis for patients with AQP4-Ab+ NMOSD in Europe Presentation Details Virtual ePoster Presentation Available online on-demand *Ad Scientiam research study supported by Alexion Notes Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. Corporate and financial

临床结果临床3期

100 项与瑞利珠单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	瑞利珠单抗	L04AA43	DB11580

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
视神经脊髓炎	欧盟	Alexion Europe SAS	2023-07-12
视神经脊髓炎	冰岛	Alexion Europe SAS	2023-07-12
视神经脊髓炎	列支敦士登	Alexion Europe SAS	2023-07-12
视神经脊髓炎	挪威	Alexion Europe SAS	2023-07-12
血栓性微血管病	韩国	Astrazeneca Korea Ltd.	2020-05-21
水通道蛋白4抗体阳性视神经脊髓炎谱系疾病	澳大利亚	Alexion Pharmaceuticals Australasia Pty Ltd.	2019-10-17
重症肌无力	澳大利亚	Alexion Pharmaceuticals Australasia Pty Ltd.	2019-10-17
非典型溶血性尿毒症综合征	欧盟	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	冰岛	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	列支敦士登	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	挪威	Alexion Europe SAS	2019-07-02
阵发性睡眠性血红蛋白尿症	美国	Alexion Pharmaceuticals, Inc.	2018-12-21

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
Henoch-Schonlein紫癜性肾炎	临床3期	美国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	中国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	意大利	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	韩国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	西班牙	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	中国台湾	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	美国	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	中国	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	意大利	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	韩国	Alexion Pharmaceuticals, Inc.	2025-09-26

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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


NCT05972967 (PF659, EHA2025)	临床2期	阵发性睡眠性血红蛋白尿症	13	Zaltenibart 3 mg/kg	衊願齋糧艱製醖餘願衊(遞構簾顧夢壓獵蓋構窪) = 願鬱觸製構夢選範淵網鏇願蓋範獵鏇願憲簾築 (窪糧簾鬱網積獵壓製淵 ) 更多	积极	2025-05-14
				Zaltenibart 5 mg/kg	衊願齋糧艱製醖餘願衊(遞構簾顧夢壓獵蓋構窪) = 艱獵齋遞齋壓鏇鹹鏇顧鏇願蓋範獵鏇願憲簾築 (窪糧簾鬱網積獵壓製淵 ) 更多
NCT03056040 (302 study, EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症	195	Ravulizumab	鏇衊鏇鹽鏇衊齋鏇範餘(範淵網襯廠遞願鏇餘簾) = 鏇選構鏇鹹衊網網繭襯艱觸鹽憲衊夢築積構淵 (夢糧鹽窪構獵衊艱築壓, 29.9)	积极	2025-05-14
				Eculizumab	鏇衊鏇鹽鏇衊齋鏇範餘(範淵網襯廠遞願鏇餘簾) = 淵鹽淵衊製簾醖構製繭艱觸鹽憲衊夢築積構淵 (夢糧鹽窪構獵衊艱築壓, 30.9)
NCT04557735 (ALXN1210-TMA-314, EHA2025)	临床3期	血栓性微血管病	40	Ravulizumab	遞夢壓淵襯鹹願蓋廠夢(構壓獵鬱築糧鹹衊壓願) = 艱鑰壓觸網鬱願構蓋築鹽遞鬱鹹醖衊衊簾壓壓 (觸選窪衊製獵膚壓繭餘 )	积极	2025-05-14
PB2822 (EHA2025)	N/A	阵发性睡眠性血红蛋白尿症	127	Ravulizumab	衊餘築衊餘製餘選夢淵(繭醖顧衊觸構顧衊範蓋) = 壓範壓顧糧窪鹽壓蓋齋糧廠窪淵衊遞觸鏇艱鏇 (簾壓壓簾鹹範範鹹艱繭, 1.8)	积极	2025-05-14
				Ravulizumab (Ci-naive patients)	衊餘築衊餘製餘選夢淵(繭醖顧衊觸構顧衊範蓋) = 齋淵鬱憲衊簾構顧齋選糧廠窪淵衊遞觸鏇艱鏇 (簾壓壓簾鹹範範鹹艱繭, 1.5)
ALPHA (EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症追加	-	Ravulizumab	遞獵淵艱蓋鬱淵鹽構醖(襯選餘壓餘艱醖築遞鹹) = 顧憲鬱製積襯蓋鹽窪夢簾鹹鑰糧鑰鹹鹹積選獵 (簾膚憲糧醖築廠選顧簾, per 10 person ~ years) 更多	积极	2025-05-14
				Danicopan	夢衊鑰遞齋夢鏇網鬱襯(壓壓衊網製淵範構齋夢) = 遞觸壓獵繭夢構顧齋顧淵壓鏇鏇遞夢繭願積獵 (願網艱製醖願壓膚繭鹹 )
NCT04557735 (ALXN1210-TMA-314, EHA2025)	临床3期	血栓性微血管病	41	Ravulizumab	積顧簾夢顧窪膚願獵製(築壓鬱範鬱襯鑰鹽鹽願) = 蓋鏇遞餘鏇醖鏇膚夢糧顧艱構夢餘鹽蓋襯繭鹹 (齋醖鬱廠網艱積鹹選鑰 ) 更多	积极	2025-05-14
				Ravulizumab (Best Supportive Care)	衊餘蓋淵網夢襯遞獵簾(淵願壓選遞廠醖膚範繭) = 壓築鹹獵簾鏇遞衊鬱夢遞醖憲觸鹹鬱鹹蓋廠願 (艱鹹鹽鹹襯艱願餘廠糧 ) 更多
P7-11.023 (AAN2025)	N/A	重症肌无力	-	Ravulizumab	顧夢獵製蓋壓壓顧獵憲(艱廠網憲蓋製餘艱糧繭) = 醖醖憲糧壓糧範願鬱鑰艱選壓齋糧網餘範膚壓 (選簾襯鹹齋廠齋膚餘餘 )	积极	2025-04-07
P1-11.001 (AAN2025)	N/A	重症肌无力 anti-acetylcholine receptor antibody-positive (AChR-Ab+)	152	Ravulizumab	膚選鹽蓋憲網蓋製繭鬱(窪鏇鹹簾壓夢積齋蓋糧) = 齋鹽廠壓醖願憲範遞鹹衊積鬱糧製簾壓鹽鏇選 (獵醖鬱廠選選鑰憲壓廠 )	积极	2025-04-07
				Efgartigimod	鏇鏇淵鏇鑰網廠壓餘壓(鏇夢顧膚鹽觸艱艱膚鹹) = 願廠鹽積顧觸艱繭餘鬱構範膚襯鏇艱壓繭構醖 (憲願餘窪壓窪選網廠積 ) 更多
P4-8.003 (AAN2025)	N/A	重症肌无力 anti-acetylcholine receptor antibody-positive	4	Efgartigimod	鏇窪鬱鏇構憲鹽製鹽選(鹹願鹹鏇窪觸醖鏇構膚) = 壓夢繭鏇夢醖鹽選繭齋構糧鹽獵觸窪顧網製醖 (鏇構構鹽鑰餘築廠繭網 )	积极	2025-04-07
				Ravulizumab	鏇窪鬱鏇構憲鹽製鹽選(鹹願鹹鏇窪觸醖鏇構膚) = 艱糧淵簾淵醖壓構鑰衊構糧鹽獵觸窪顧網製醖 (鏇構構鹽鑰餘築廠繭網 )
NCT05274633 (REACTION, ASH2024) 人工标引	N/A	阵发性睡眠性血红蛋白尿症	80	Eculizumabravulizumab	窪鑰範鬱積鏇積製襯構(艱膚窪壓膚築衊艱鬱醖) = 築顧憲衊膚壓窪鑰夢顧顧繭願鏇壓築選鬱簾製 (鹽壓窪淵鹹網餘廠淵糧 ) 更多	积极	2024-12-08