A Multicenter, Single Arm, Open Label Biopsy Study to Evaluate Structural and Functional Changes in Kidneys of Adult Patients With IgA Nephropathy Receiving Iptacopan on Top of Supportive Care

A study to investigate the impact of iptacopan treatment on the underlying immunopathology in patients with IgAN by assessing changes in key clinical and molecular markers from baseline to 9 months. The study aims to provide insights into the treatment's systemic and kidney-specific aspects by quantifying the change in mesangial C3c containing fragments deposition, as an indicator of complement activation, and evaluating a variety of biomarkers related to kidney function, damage, and disease progression, including but not limited to Oxford MEST-C score.

开始日期2025-02-24

申办/合作机构

Novartis Pharmaceuticals Corp.

100 项与盐酸伊普可泮相关的临床结果

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100 项与盐酸伊普可泮相关的转化医学

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100 项与盐酸伊普可泮相关的专利（医药）

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项与盐酸伊普可泮相关的文献（医药）

2025-04-01·KIDNEY INTERNATIONAL

Treatment of patients with IgA nephropathy: a call for a new paradigm

Review

作者: Floege, Jürgen ; Barratt, Jonathan ; Rovin, Brad ; Bernier-Jean, Amelie

IgA nephropathy (IgAN), the world's most common primary glomerular disease, carries a significant lifetime risk for kidney failure as well as an enormous socioeconomic burden. In the past, studies in patients with IgAN largely focused on optimizing so-called supportive care, that is, blockade of the renin-angiotensin system, blood pressure control, and lifestyle modifications. The effectiveness of immunosuppressive measures, particularly high-dose corticosteroid therapy, has been reported variably, but there is considerable evidence for an increase in serious adverse effects with such therapies. This disappointing situation has changed dramatically with a better understanding of the pathogenesis of IgAN, and with regulatory agencies accepting changes in proteinuria and the estimated glomerular filtration rate loss or slope over 2 to 3 years as surrogate outcome markers. A multitude of new therapies are now being evaluated in IgAN, and several drugs, such as sodium-glucose transporter-2 inhibitors, sparsentan (a dual endothelin-1 and angiotensin II receptor blocker), nefecon (a targeted release formulation of budesonide), and iptacopan (a complement factor B inhibitor), have been approved, with more to come in the next few years. In this review, we propose a new treatment paradigm that combines therapies with different mechanisms of action to target the immune components and the chronic kidney disease components of IgAN in parallel to preserve long-term kidney survival.

2025-02-06·NEW ENGLAND JOURNAL OF MEDICINE

Alternative Complement Pathway Inhibition with Iptacopan in IgA Nephropathy

Article

作者: Hach, Thomas ; Guerard, Nicolas ; Renfurm, Ronny ; Kollins, Dmitrij ; Rovin, Brad ; Perkovic, Vlado ; Papachristofi, Olympia ; Kashihara, Naoki ; Trimarchi, Hernán ; Barratt, Jonathan ; Jacinto-Sanders, Severina ; Rizk, Dana V. ; Merkel, Tobias ; Maes, Bart ; Zhang, Hong

BACKGROUND:

The alternative complement pathway plays a key role in the pathogenesis of IgA nephropathy. Iptacopan specifically binds to factor B and inhibits the alternative pathway.

METHODS:

In this phase 3, double-blind, randomized, placebo-controlled trial, we enrolled adults with biopsy-confirmed IgA nephropathy and proteinuria with a 24-hour urinary protein-to-creatinine ratio of 1 or higher (with protein and creatinine both measured in grams) despite optimized supportive therapy. Patients were randomly assigned, in a 1:1 ratio, to receive oral iptacopan (200 mg) or placebo twice daily for 24 months while continuing to receive supportive therapy. The primary objective of this prespecified interim analysis was to assess the efficacy of iptacopan as compared with that of placebo in reducing proteinuria at month 9; the primary end point was the change from baseline in the 24-hour urinary protein-to-creatinine ratio at month 9. The proportion of patients who had a 24-hour urinary protein-to-creatinine ratio of less than 1 at month 9 without receiving rescue or alternative medication or undergoing kidney-replacement therapy (dialysis or transplantation) was a secondary end point. Safety was also assessed. The effect of iptacopan on kidney function will be assessed at the end of the 2-year double-blind treatment period.

RESULTS:

The main trial population included 222 patients in the iptacopan group and 221 in the placebo group. The interim efficacy analysis included the first 250 patients who underwent randomization in the main trial population (125 patients in each group) and who remained in the trial until month 9 or discontinued the trial by month 9. Safety was assessed in all the patients in the main trial population. At month 9, the adjusted geometric mean 24-hour urinary protein-to-creatinine ratio was 38.3% (95% confidence interval, 26.0 to 48.6; two-sided P<0.001) lower with iptacopan than with placebo. The reduction in proteinuria was supported by consistent results in secondary end point analyses. There were no unexpected safety findings with iptacopan. The incidence of adverse events that occurred during the treatment period was similar in the two groups; most events were mild to moderate in severity and reversible. No increased risk of infection was observed.

CONCLUSIONS:

Among patients with IgA nephropathy, treatment with iptacopan resulted in a significant and clinically meaningful reduction in proteinuria as compared with placebo. (Funded by Novartis; APPLAUSE-IgAN ClinicalTrials.gov number, NCT04578834.).

2025-02-01·INFLAMMOPHARMACOLOGY

Inhibition of alternative complement system and prolyl hydroxylase ameliorates anaemia of inflammation

Article

作者: Jain, Mukul R ; Dodiya, Harshad S ; Kshirsagar, Samadhan G. ; Savsani, Hardikkumar H. ; Patel, Maulik S ; Patel, Vishal J ; Jain, Mukul R. ; Dodiya, Harshad S. ; Joharapurkar, Amit A. ; Rakhasiya, Milan H ; Kshirsagar, Samadhan G ; Patel, Maulik S. ; Savsani, Hardikkumar H ; Rakhasiya, Milan H. ; Joharapurkar, Amit A ; Patel, Vishal J.

Chronic diseases associated with inflammation cause early destruction of RBCs. Complement system, part of innate immunity, is involved in such RBC destruction. Persistent inflammation causes kidney injury, leading to reduced erythropoietin release and functional iron deficiency, causing anaemia. We have investigated effect of iptacopan, a factor B inhibitor, and desidustat, a prolyl hydroxylase inhibitor in anaemia induced by peptidoglycan polysaccharide (PGPS) treatment in rats. Inflammation, haemolysis and its diagnostic markers (LDH, total bilirubin, and RBC lifespan) were evaluated after three days of PGPS challenge. Haemoglobin, RBC, iron homeostasis, and RBC destruction were evaluated fourteen days after PGPS challenge. Desidustat (15 mg/kg) and iptacopan (20 mg/kg) were given along with PGPS and continued for two weeks. Iptacopan and its combination with desidustat prevented LDH, total bilirubin, complement protein-C3a and haemolysis. Combination treatment caused an early normalization of haemoglobin and RBC. Combination also reduced WBC, alkaline phosphatase, aspartate aminotransferase, and rat paw volume. Serum iron was increased by desidustat and its combination treatment. Spleen weight, tissue iron, and serum hepcidin were reduced by combination treatment. Effect of desidustat alone was prominent on iron (serum and tissue) and hepcidin. Thus, combination of iptacopan and desidustat can be a potentially useful therapeutic option for treatment of anaemia of inflammation.

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项与盐酸伊普可泮相关的新闻（医药）

2025-03-25

·蒲公英Ouryao

Paul's Insight｜解读国际法规药事(03.17-03.23)

◆ ◆ ◆ ◆ Paul's Insight 2025年3月17日-3月23日 ◆ ◆ ◆ ◆ 本期药事动态（3月17日至23日）聚焦美国食品药品监督管理局（FDA）、美国注射剂协会（PDA）、欧洲药品管理局（EMA）、欧洲药品质量管理局（EDQM）、英国药品和医疗产品监管局（MHRA）、世界卫生组织（WHO）、国际人用药品注册技术协调理事会（ICH）以及国际制药工程协会（ISPE）近期发布的关键信息，通过一条“创新驱动，安全至上”的主线，展现全球药事监管机构在确保药品质量和患者安全的同时，积极拥抱创新、优化监管流程的努力。美国FDA 严守质量红线，拥抱治疗创新美国FDA近期动作频频，既展现了其对药品生产质量的严格把控，也体现了对创新疗法的积极接纳。 1. 批准诺华重磅药物 3月20日， FDA 批准了诺华公司（Novartis）的Fabhalta（iptacopan）用于治疗C3肾小球病（C3G），这是一种罕见的肾脏疾病。这是FDA首次批准用于治疗C3G的药物，也是Fabhalta继2023年获批用于阵发性睡眠性血红蛋白尿症（PNH）和2024年获批用于原发性IgA肾病（IgAN）之后的第三个适应症。Fabhalta的获批为C3G患者带来了首个针对疾病根源的治疗选择，有望成为新的治疗标准。图：FDA批准诺华重磅药物（参考：https://www.fda.gov/news-events/press-announcements/fda-roundup-march-21-2025） 2.创新疗法获批：Pembrolizumab的新里程碑 3月19日，FDA批准默沙东（MSD）的磅PD-1抑制剂Keytruda（pembrolizumab），用于局部晚期不可切除或转移性人表皮生长因子受体2（HER2）阴性胃或胃食管连接部（GEJ）腺癌成人患者的一线治疗。Keytruda是一种人源化的抗PD-1单克隆抗体，可以阻断PD-1与其配体PD-L1、PD-L2的结合，进而激活T淋巴细胞。此作用可能增强体内免疫系统侦测与抵抗肿瘤细胞的能力。 2014年9月，Keytruda首次获FDA批准治疗晚期黑色素瘤，并在后续获批作为一线疗法治疗转移性NSCLC患者。在中国，Keytruda也已获批多项适应症，涵盖黑色素瘤、非小细胞肺癌、头颈部鳞状细胞癌、结直肠癌、食管癌、肝癌等。图：FDA批准默沙东（MSD）PD-1抑制剂（参考：https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-her2-positive-gastric-or-gastroesophageal-junction-adenocarcinoma） 3. 质量警钟长鸣：FDA多封警告信 3月18日，FDA针对分布于多个国家的多家企业发布了一系列警告信，再次强调了其对药品生产质量的高度关注和零容忍态度。 Vegewax Candleworx Ltd：该加拿大公司因生产过程中的严重不合规问题收到警告信，主要涉及生产环境、操作流程和质量控制措施的缺陷，特别是设备维护和卫生标准执行不力。图：FDA给加拿大一家公司的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/vegewax-candleworx-ltd-675099-06142024） Aspen Pharmacare Holdings Limited：该南非药企为无菌制剂的CMO工厂。FDA指出该公司在质量标准和无菌保障方面存在多项违规行为，包括未制定适当的杂质限度、无菌操作不当、烟雾研究和洁净室设计不足、计算机系统控制不当以及环境监控不充分等问题。图：FDA给南非无菌制剂CMO工厂的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/aspen-pharmacare-holdings-limited-701671-02242025） Neva Global Grup Sanayi İc ve Dis Ticaret Anonim Şirketi：该土耳其企业在生产、质量控制等环节被发现存在明显缺陷，包括检测流程不严、工艺验证不充分、稳定性研究不足以及质量部门未能履职等问题。图：FDA给土耳其一家公司的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/neva-global-grup-sanayi-ic-ve-dis-ticaret-anonim-sirketi-699000-03122025） Rite-Kem Incorporated： FDA对该美国本土公司发出警告信，详细说明了其在药品生产过程中未能充分执行GMP，在产品检验、工艺验证、投诉管理以及员工培训方面存在严重漏洞。图：FDA给美国本土一家公司的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/rite-kem-incorporated-697272-01282025） International Laboratories Corp：该泰国检验实验室因产品测试、批次放行和伪造实验室记录等问题收到警告信，表明其质量管理体系存在缺陷，可能导致药品安全隐患。在检查中，FDA调查员发现了多起伪造实验室记录的例子。在检查期间，其实验室经理表示，公司伪造了几项未进行的实验室调查。一位高级质量保证经理也证实，实验室调查记录是为准备 FDA 检查而伪造的。图：FDA给泰国检验实验室的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/international-laboratories-corp-698522-03102025） Advanced Pharmaceutical Technology：该公司为美国一家无菌生产企业。FDA指出该公司在无菌保障和质量管理上存在系统性问题。如FDA指出，公司缺乏适当的控制措施来确保无菌药品的无菌性。例如，公司依赖终端灭菌工艺实现无菌，但没有监测灭菌前的生物复核。此外，公司未能按照SOP的要求建立适当的系统来监测环境条件（包括执行频率），以确保生产区域适合无菌药品生产。图：FDA给美国一家无菌生产企业的警告信（参考：https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/advanced-pharmaceutical-technology-692576-03142025） 4.FDA内部管理面临挑战：工作人员近日被要求返回办公室多家媒体报道，由于特朗普政府取消了许多联邦雇员的远程办公权利，FDA工作人员近日被要求返回办公室。一些返岗员工表示，他们面临着进办公室要排长队、办公室短缺等问题。同时，上个月被解雇的 FDA 试用期员工已复职，但他们将继续享受带薪行政假，直至另行通知。FDA面临的内部管理挑战，可能导致机构内部协作和信息传递出现混乱。这提醒监管机构在关注外部监管的同时，也需不断优化内部管理，以确保监管工作的顺利进行。图：FDA工作人员近日被要求返回办公室（参考：https://www.raps.org/News-and-Articles/News-Articles/2025/3/FDA-s-return-to-office-chaos,-probationary-workers）美国PDA 前瞻性思考全球监管趋同美国注射剂协会（PDA）近期发表文章，深入探讨了全球药品监管趋同的可能性及其对制药行业的深远影响。全球监管趋同的探索与思考（2025年3月17日）背景与意义：文章回顾了近年来各国监管机构在药品审批及合规要求方面不断趋同的趋势，并指出其对于提升药品安全性和加速新药上市具有重要意义。关键挑战与机遇：文章分析了实现全球监管趋同过程中面临的挑战，如法律法规差异、文化与监管传统不同以及经济发展水平的差距，同时也探讨了其为制药企业带来的机遇，例如降低成本、加速国际合作等。未来展望与建议： PDA提出了加强跨国监管机构合作、推动数据共享、采用风险导向监管模式以及鼓励国际标准制定的建议。行业影响：文章认为，实现监管趋同将有助于提高全球市场透明度，降低企业合规负担，并为患者提供更及时、有效的治疗选择。图：PDA关于前瞻性思考全球监管趋同（参考：https://www.pda.org/pda-letter-portal/home/full-article/global-regulatory-convergence-what-if） PDA通过其前瞻性的分析，强调了国际合作在推动全球药品监管进程中的重要性，并为行业提供了应对监管变化的策略思考。欧洲EMA 拥抱人工智能，擘画监管蓝图欧洲药品管理局（EMA）近期在拥抱技术创新和制定未来监管战略方面展现出积极的姿态。3月20日， EMA正式认可首个利用人工智能技术辅助诊断炎症性肝病的工具。该工具名为AIM-NASH，可帮助病理学家分析肝活检扫描结果，以确定临床试验中 MASH（代谢功能障碍相关脂肪性肝炎；以前称为非酒精性脂肪性肝炎 NASH）的严重程度。这标志着监管机构对新兴技术的积极接纳，预示着未来人工智能在医疗影像和诊断领域将有更广阔的应用前景。EMA强调将持续关注人工智能在医疗领域的安全性和有效性。图：EMA 批准首个人工智能工具用于诊断活检样本中的炎症性肝病 (MASH) （参考：https://www.ema.europa.eu/en/news/ema-qualifies-first-artificial-intelligence-tool-diagnose-inflammatory-liver-disease-mash-biopsy-samples） EMA这一举措表明，欧洲监管机构正积极适应医药科技的发展趋势，通过拥抱创新技术和优化监管战略，致力于在确保药品和医疗器械安全有效的前提下，加速科技成果转化和医疗服务创新。欧洲EDQM 聚焦国际合作，提升质量标准欧洲药品质量管理局（EDQM）近期发布的一则公告显示着其在推动国际合作和提升药品质量标准方面的新动向。3月19日， EDQM宣布将召开一次国际会议，重点讨论其CEP（欧洲药典适用性证书）程序。会议旨在为制药企业、监管机构和专家提供一个平台，共同探讨如何利用CEP程序进一步提高药品质量监管水平，并推动国际间的技术合作与信息共享。此次会议的召开将有助于加强各方对CEP程序的认识，推动制定更科学、有效的质量评估标准。EDQM通过组织国际会议，积极促进全球范围内对药品质量标准的理解和应用，进一步提升欧洲乃至全球的药品质量安全水平。图：EDQM：聚焦国际合作，提升质量标准（参考：https://www.edqm.eu/en/-/save-the-date-international-conference-on-the-edqm-s-cep-procedure）英国MHRA 保障产品安全，拥抱分散制造英国药品和医疗产品管理局（MHRA）近期在产品召回和探索新型制造模式方面展现出积极的监管行动。 1.产品召回警示：保障患者安全苏州 Surgicare 一次性宫腔镜鞘召回通知：3月22日，MHRA发布召回通知，针对苏州 Surgicare 公司（Suzhou Surgicare Medical Technology Ltd）生产的一次性宫腔镜鞘，原因是其CE认证已被撤销，存在合规性问题，可能影响产品安全性和临床使用效果。这体现了MHRA对医疗器械质量和患者安全的高度重视。图：苏州 Surgicare 一次性宫腔镜鞘召回通知（参考：https://www.gov.uk/drug-device-alerts/suzhou-surgicare-disposable-hysteroscopy-sheath-recall-due-to-withdrawn-ce-certificate-dsi-slash-2025-slash-001） 2.前瞻性监管：探索分散式制造模式 3月19日，MHRA在其官网更新了“分散制造（Decentralised Manufacture Hub）”集合页面，汇总了与分散式制造模式相关的政策信息、监管指南及动态更新。此举旨在推动分散式生产模式在药品制造领域的应用，提高供应链的韧性、降低风险并加快应急响应。MHRA通过这一平台，向行业传递最新的监管思路和支持措施，确保新兴制造模式在保证药品安全与质量的前提下顺利融入市场。MHRA的行动既体现了对现有产品质量问题的及时响应，也展现了其对未来药品生产模式的积极探索和监管适应性调整。图：MHRA探索分散式制造模式（参考：https://www.gov.uk/drug-device-alerts/suzhou-surgicare-disposable-hysteroscopy-sheath-recall-due-to-withdrawn-ce-certificate-dsi-slash-2025-slash-001） WHO 优化预认证流程，加速药品可及性世界卫生组织（WHO）近期宣布对其药品预认证项目（PQTmed）的简略预认证途径进行扩展和优化，旨在加速高质量药品的预认证流程，提高全球公众获得安全、有效药品的速度。WHO的这一举措将对全球制药行业产生一定影响，推动各方在确保产品质量和安全的同时，更快响应市场需求，提高药品的可及性。图：WHO优化预认证流程（参考：https://extranet.who.int/prequal/news/pqtmed-expanded-abridged-prequalification-pathways） ICH 推进技术规范，统一申报标准国际人用药品注册技术协调理事会（ICH）近期在推进国际药品申报标准和技术规范更新方面取得了进展。 eCTD v4.0 Q&A v1.9达到ICH流程第四步（3月20日）背景与意义： eCTD格式作为全球药品申报的标准化工具，其问答指南的更新对于确保提交文件符合最新技术和监管要求至关重要，有望在全球范围内推动申报效率的提升。关键内容与后续步骤：更新后的问答文件进入专家审查和多方协商阶段，将广泛征求监管机构、制药企业及其他利益相关者的意见。图：eCTD v4.0 Q&A v1.9 已达到 ICH 流程的第 4 步（参考：https://www.ich.org/news/ectd-v40-qa-v19-reaches-step-4-ich-process） ICH M11更新演示文稿上线（3月20日）更新背景与目的： ICH M11指南文件的技术规范更新旨在反映最新科学进展和监管实践经验，此次演示文稿详细说明了更新范围、修订动因及预期效果。图：ICH M11更新演示文稿上线（参考：https://www.ich.org/news/ich-m11-updated-technical-specification-step-2-presentation-now-available-ich-website） ICH的持续努力在于优化和统一全球药品监管要求，提升申报效率，并最终确保药品质量和安全。 ISPE 应对数字转型，展望制药就业未来国际制药工程协会（ISPE）近期关注美国制药制造业在数字化转型和自动化技术普及背景下，如何确保就业岗位具备持续竞争力和适应未来发展的能力。行业现状与转型需求：文章指出，新技术在制药生产流程中的应用对现有工作岗位的技能要求提出了更高标准，监管环境的变化和全球化竞争也使得企业必须在技术创新与人才培养之间找到平衡。技能升级与人才培养：文章强调了员工技能升级和跨领域培训的重要性，建议制药企业重新规划人才发展战略，培养新型复合型人才。监管与政策导向的影响：文章分析了不断演进的监管要求对制药制造业的影响，并呼吁监管部门与企业之间加强沟通与协作，共同制定支持创新又兼顾安全的政策措施。未来展望与建议：文章提出，未来的制药制造业将呈现岗位结构调整、技能需求升级和工作模式转变的综合特征，建议企业和监管机构提前布局，通过技术革新、流程再造和人才储备，构建一个既能应对未来挑战又能抓住新兴机遇的行业生态系统。 ISPE的文章为美国制药制造业未来的就业趋势提供了前瞻性分析，并为如何通过技术创新与人才培养实现行业可持续发展指明了方向。图：ISPE应对数字转型，展望制药就业未来（参考：https://ispe.org/pharmaceutical-engineering/ispeak/futureproofing-us-pharma-manufacturing-jobs） 01 综上所述，全球各大药事监管和行业机构近期的一系列举措、思考，无不体现着“创新驱动，安全至上”的核心理念。从对创新疗法的积极审批到对生产质量的严格把控，从拥抱人工智能等前沿技术到前瞻性地探索未来监管模式，全球药事监管体系正不断进化，以适应快速发展的医药行业和日益增长的健康需求。通过加强国际合作、优化监管流程、提升技术标准，各国监管机构正携手共进，共同构建一个更加安全、高效、可靠的全球药品监管体系，最终目标是护佑全人类的健康福祉。 END 声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。近期活动课程推荐线上 3月26日深度解析多样化抗体平台，突破抗体工艺瓶颈线下 3月28日邀请函 | 春启未来：创新药出海与工艺革新论坛线下 4月11日 4月上海丨智药领航行—医药质量数字化赋能研讨会

申请上市上市批准

2025-03-25

·PRNewswire

Novartis to present new data at AAN, including seven-year disability outcomes and safety analysis of Kesimpta® in people with relapsing multiple sclerosis

Long-term disability and safety data from ALITHIOS open-label extension study on continuous treatment with Kesimpta vs. later switch from teriflunomide in relapsing multiple sclerosis (RMS) patients to be presented Additional presentations highlight the Novartis neuroscience pipeline, including remibrutinib for RMS and iptacopan for generalized myasthenia gravis EAST HANOVER, N.J., March 25, 2025 /PRNewswire/ -- Novartis will present data from studies across its neuroscience portfolio, including seven-year disability outcomes and safety data from the ALITHIOS open-label extension trial of Kesimpta® (ofatumumab) in people with relapsing multiple sclerosis (RMS), at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego from April 5-9, 2025. Additional data on Kesimpta and pipeline assets including remibrutinib and iptacopan will also be highlighted. "We look forward to sharing several clinical and real-world studies on Kesimpta in people living with RMS as well as additional studies across our neuroscience portfolio," said Norman Putzki, M.D., Ph.D., Development Unit Head, Neuroscience & Gene Therapy, Development, Novartis. "As always, we are excited to attend the AAN annual meeting and discuss the latest developments in neuroscience as we work to address some of the most pressing challenges for people living with neurological conditions." Data to be presented at AAN include: Product Information For full prescribing information, including approved indications and important safety information about marketed products, please visit . Novartis in Neuroscience At Novartis, we have been tackling neurological conditions for more than 80 years, launching transformative treatments which have made meaningful differences to millions of people worldwide now and in the future. We continue to collaborate on industry-leading treatments in multiple sclerosis, neuroimmunology, neurodegeneration and neuromuscular/rare diseases. Disclaimer This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and and connect with us on LinkedIn, LinkedIn US, Facebook, X/Twitter, X/Twitter US and Instagram. # # # SOURCE Novartis Pharmaceuticals Corporation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床研究基因疗法

2025-03-25

·PMLiVE

Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G

Novartis’ oral Fabhalta (iptacopan) has been approved by the US Food and Drug Administration (FDA) as the first treatment for the ultra-rare kidney disease complement 3 glomerulopathy (C3G). Every year, approximately one to two people per million worldwide are diagnosed with C3G, in which deposits of C3 protein build up in the kidney glomeruli, the body’s network of blood vessels designed to filter waste and remove extra fluids from the blood. This causes inflammation and glomerular damage that results in proteinuria (protein in urine), haematuria (blood in urine) and reduced kidney function, with about half of C3G patients progressing to kidney failure within ten years of diagnosis. Fabhalta, which has been specifically authorised to reduce proteinuria in adults with C3G, selectively targets what is thought to be the underlying cause of the disease. The drug is already approved in the US to treat patients with immunoglobulin A nephropathy, another rare kidney disease, and the rare blood disorder paroxysmal nocturnal haemoglobinuria. The FDA’s latest decision was based on positive result from the phase 3 APPEAR-C3G study, which showed that patients being treated with Fabhalta alongside supportive care achieved a statistically significant and clinically meaningful 35.1% reduction in proteinuria at six months compared to those randomised to receive placebo. Data on the secondary endpoint of estimated glomerular filtration rate, a measure of kidney function, also showed improvements over six months with Fabhalta compared to placebo, and Novartis’ therapy demonstrated a favourable safety profile with no new safety signals observed. APPEAR-C3G study co-investigator, Carla Nester, said: “C3G is a debilitating disease often affecting young people, impacting many aspects of their physical and emotional health, and our previous treatment options came with significant challenges. “This approval of Fabhalta is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients.” The decision comes less than a month after Fabhalta was recommended by the European Medicines Agency’s human medicines committee to treat adults with C3G. The European Commission will now review the Committee for Medicinal Products for Human Use’s recommendation as it makes a decision on Fabhalta in this indication.

临床结果上市批准临床3期

100 项与盐酸伊普可泮相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	盐酸伊普可泮	-	DB16200

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
C3肾小球病	美国	Novartis Pharmaceuticals Corp.	2025-03-20
免疫球蛋白a肾病	美国	Novartis Pharmaceuticals Corp.	2024-08-07
阵发性睡眠性血红蛋白尿症	美国	Novartis Pharmaceuticals Corp.	2023-12-05

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
补体因子H缺乏症	申请上市	欧盟	Novartis Europharm Ltd.	2025-02-27
重症肌无力	临床3期	美国	Novartis Pharmaceuticals Corp.	2024-07-31
重症肌无力	临床3期	美国	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	中国	Novartis Pharmaceuticals Corp.	2024-07-31
重症肌无力	临床3期	日本	Novartis Pharmaceuticals Corp.	2024-07-31
重症肌无力	临床3期	日本	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	丹麦	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	德国	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	希腊	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	希腊	Novartis Pharmaceuticals Corp.	2024-07-31

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临床结果

适应症

分期

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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


NCT04817618 (APPEAR-C3G, FDA_CDER) 人工标引	临床3期	C3肾小球病	74	FABHALTA 200 mg	壓衊窪淵願構顧艱願繭(顧糧積襯鏇鏇齋糧鹽網) = 艱鹽廠鏇構築鑰構窪顧廠廠鹽遞淵齋築選構窪 (鏇製憲窪獵齋築膚淵襯, 0.57 ~ 0.85) 更多	积极	2025-03-20
				Placebo	壓衊窪淵願構顧艱願繭(顧糧積襯鏇鏇齋糧鹽網) = 觸選鹽齋願憲餘醖範觸廠廠鹽遞淵齋築選構窪 (鏇製憲窪獵齋築膚淵襯, 0.88 ~ 1.31) 更多
NCT04820530 \| NCT04558918 (APPOINT-PNH, Pubmed) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	135	iptacopan (C5i-experienced patients)	齋夢膚鹽餘鹽遞顧範壓(遞鏇窪齋醖製鹹淵鏇蓋) = 鬱糧鏇齋遞齋積網觸範鹹夢衊艱膚選壓積網網 (膚艱遞製膚衊鏇餘鬱獵 ) 更多	积极	2025-01-07
				Placebo (C5i-experienced patients)	齋夢膚鹽餘鹽遞顧範壓(遞鏇窪齋醖製鹹淵鏇蓋) = 窪壓餘淵選窪願獵獵觸鹹夢衊艱膚選壓積網網 (膚艱遞製膚衊鏇餘鬱獵 ) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Iptacopan monotherapy 200 mg twice daily	鹹壓鏇繭遞壓範衊鏇觸(觸構鑰憲製網齋齋築觸) = 壓壓餘齋鏇鹹製築艱糧遞憲餘醖遞選夢簾壓製 (淵淵鏇範積醖衊鏇網糧, 87.4) 更多	-	2024-12-09
				Eculizumab	鹹壓鏇繭遞壓範衊鏇觸(觸構鑰憲製網齋齋築觸) = 艱鏇遞醖製襯範夢獵鬱遞憲餘醖遞選夢簾壓製 (淵淵鏇範積醖衊鏇網糧, 77.2) 更多
ASH2024	N/A	冷凝集素病	-	Iptacopan 200 mg	廠艱膚膚憲製窪鏇廠築(襯鬱願鹽繭鏇鬱夢憲醖) = Eight pts (80%) experienced ≥1 treatment-emergent adverse event (TEAE), most of which were mild in severity. Two pts discontinued treatment because of TEAEs (1 had increased alanine aminotransferase and aspartate aminotransferase [suspected to be treatment related]; 1 had recurrent breast cancer [not suspected to be treatment related]). Two pts had serious adverse events (1 had increased blood creatinine and acute kidney injury; 1 had spinal fracture), but none were suspected to be treatment related. 淵鏇衊淵繭淵積衊鏇網 (餘襯醖願艱網醖淵鏇繭 )	-	2024-12-08
NCT04578834 (APPLAUSE-IgAN, FDA_CDER) 人工标引	临床3期	免疫球蛋白a肾病	250	FABHALTA	選艱鬱淵網廠獵艱簾簾(構顧鹽選鑰餘鬱繭觸選) = 範築壓廠鬱蓋繭遞築觸範築淵鑰蓋蓋獵淵襯選 (築鑰築製獵願廠餘鑰憲, 36 ~ 51) 更多	积极	2024-08-07
				Placebo	選艱鬱淵網廠獵艱簾簾(構顧鹽選鑰餘鬱繭觸選) = 襯顧膚襯衊衊憲餘網鏇範築淵鑰蓋蓋獵淵襯選 (築鑰築製獵願廠餘鑰憲, -5 ~ 21) 更多
NCT04154787 (CTgov)	临床2期	特发性膜性肾病	37	Rituximab	選遞構範糧觸憲夢獵衊(觸鹹鹹顧艱構製繭鏇窪) = 衊夢遞鏇觸繭壓製鬱鬱醖鏇獵醖鬱艱糧鬱憲糧 (膚蓋築襯鑰鹽憲淵夢憲, 夢衊夢襯淵鹹夢齋糧選 ~ 艱蓋選壓壓鹹憲觸淵夢) 更多	-	2024-07-05
NCT04817618 (APPEAR-C3G, PRNewswire 1 \| PRNewswire 2) 人工标引	临床3期	C3肾小球病	-	Fabhalta (iptacopan)	窪鑰膚製鏇糧鏇範鹽選(簾遞膚壓鹹鑰壓壓鏇醖) = 膚顧鏇繭壓淵網築糧築積鬱簾憲願壓簾鏇鑰繭 (艱選憲網獵襯觸餘憲繭 )	积极	2024-05-25
				Placebo	-
NCT04558918 \| NCT03500549 (APPLY-PNH \| PEGASUS, EHA2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症维持	103	Iptacopan	餘壓遞窪襯繭艱憲範醖(構淵襯憲遞積願鑰簾衊) = 醖觸糧製築觸製襯艱簾簾餘鏇夢願醖繭醖餘襯 (壓選積構築鹽願淵鹽選 ) 更多	积极	2024-05-14
				Pegcetacoplan	餘壓遞窪襯繭艱憲範醖(構淵襯憲遞積願鑰簾衊) = 膚觸衊鏇壓蓋繭糧壓觸簾餘鏇夢願醖繭醖餘襯 (壓選積構築鹽願淵鹽選 )
NCT04820530 (APPOINT-PNH, EHA2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	40	Iptacopan 200 mg	淵壓餘鏇醖顧遞選夢顧(衊鏇鑰鏇選夢蓋鑰鑰繭) = 鑰糧簾淵鑰壓遞醖鹽廠窪網淵齋繭選憲選範艱 (鏇蓋壓鑰艱鏇壓衊遞壓 ) 更多	积极	2024-05-14
NCT04558918 (APPLY-PNH, EHA2024)	临床3期	阵发性睡眠性血红蛋白尿症 C3 fragment deposition	95	Iptacopan 200 mg twice daily	鏇遞築構獵壓鹹襯醖鏇(獵壓選觸淵鬱顧鬱壓鏇) = 遞鏇憲鏇繭夢繭觸繭積網築選餘遞齋鏇製範蓋 (構膚獵廠醖窪夢夢製鹹, -16.1%) 更多	积极	2024-05-14
				Anti-C5 inhibitors	鏇遞築構獵壓鹹襯醖鏇(獵壓選觸淵鬱顧鬱壓鏇) = 廠繭餘鑰窪積衊糧積觸網築選餘遞齋鏇製範蓋 (構膚獵廠醖窪夢夢製鹹, -16.9%) 更多