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.

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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.

400

项与盐酸伊普可泮相关的新闻（医药）

16 小时之前

·AIDD Pro

【第一百七十期】AI+药物研发领域一周资讯

前言 AIDD Pro 根据国内外各大网站以及人工智能药物设计主流新闻网站及公众号，从 AIDD会议、AIDD招聘，重大科研进展、行业动态、最新报告发布等角度，分析挖掘了每周人工智能辅助药物设计领域所发生的、对领域技术发展产生重大推动作用的事件，旨在帮助 AIDD领域研究人员和业内人士及时追踪最新科研动态、洞察前沿热点。如果您觉得符合以上要求的内容我们有遗漏或者更好建议，欢迎后台留言。科研进展2025年3月21日【BET抑制剂】ACS Med. Chem. Lett. | 基于易于处理的三环支架的高效BET抑制剂的发现2025年3月21日【SARS-CoV-2】J. Phys. Chem. Lett. | 利用分子模拟技术应对SARS-CoV-2大流行威胁的思考2025年3月20日【药物发现】J. Chem. Inf. Model. | 利用诱导极化进行药物发现：利用最小特征有效预测IC502025年3月20日【大语言模型】J. Chem. Inf. Model. | 大语言模型在药物致骨毒性预测中的应用2025年3月19日【配体】ACS Med. Chem. Lett. | Von Hippel-Lindau肿瘤抑制蛋白（VHL）Cys77共价配体的发现2025年3月19日【无监督学习】J. Chem. Theory Comput. | 加权集成模拟过程中进度坐标的无监督学习：在NTL9蛋白折叠中的应用具体信息，请滑动下方文字 1.【BET抑制剂】溴结构域和额外末端结构域（BET）蛋白家族是一类表观遗传读取蛋白，能够识别组蛋白尾部的 N-乙酰化赖氨酸残基，在基因表达和细胞转录中发挥着关键作用。选择性抑制含溴结构域蛋白（BRDs）会破坏关键癌基因的转录。在过去十年中，开发用于治疗血液系统恶性肿瘤和实体瘤的小分子 BET 抑制剂引起了极大的关注。在此，我们报告了一种三嗪并吲哚骨架的开发，该骨架能够抑制含溴结构域蛋白 4（BRD4），其中二甲基异噁唑或二甲基三唑取代基作为 N-乙酰化赖氨酸残基的化学模拟物。对母体骨架进行衍生化得到了先导化合物，该化合物对 BRD4-BD1 显示出低纳摩尔亲和力，并具有良好的物理化学性质和体外稳定性。链接网址：https://pubs.acs.org/doi/10.1021/acsmedchemlett.4c00621 DOI：https://doi.org/10.1021/acsmedchemlett.4c00621 2.【SARS-CoV-2】分子模拟对了解SARS-CoV-2病毒的生命周期具有重要作用，有助于设计和开发抗病毒药物和COVID诊断测试。在这里，我们讨论了这些模拟提供的见解和所涉及的挑战，重点是SARS-CoV-2主要蛋白酶（Mpro）和刺突糖蛋白。Mpro是抗病毒药物的主要靶标，而刺突糖蛋白是疫苗设计的靶标。最后，我们反思了这次大流行给模拟社区带来的教训。国际研究界的数据共享举措和合作有助于增进知识，应以此为基础，帮助应对未来的大流行病和抗微生物药物耐药性等其他全球挑战。链接网址：https://pubs.acs.org/10.1021/acs.jpclett.4c03654 DOI：https://doi.org/10.1021/acs.jpclett.4c03654 3.【药物发现】在这里，我们使用分子内每个原子类型（H、C、N、O、s等）的原子杂交频率（s、sp、sp2和sp3）来预测类药物分子的ic50，重点关注靶向凝血酶、雌激素受体α和磷酸二酯酶5A蛋白的化合物。神经网络和随机森林模型仅使用19个特征产生高相关系数（R2）和低均方误差（MSE）。原子杂化以前已被用于计算分子极化率使用一个简单的经验模型(米勒等。江淮1979)。我们证明原子杂化也可以用来准确地预测这些分子的分子极化率。结果表明了诱导极化在蛋白质与配体结合中的重要性。此外，不同靶蛋白的R2和MSE的变化表明，诱导极化对不同靶蛋白结合能的贡献是不同的。链接网址：https://pubs.acs.org/doi/10.1021/acs.jcim.5c00076 DOI：https://doi.org/10.1021/acs.jcim.5c00076 4.【大语言模型】药物性骨毒性是指某些药物对骨骼系统产生的有害作用，具有重大的安全风险。这些毒性作用在临床实践、药物开发和环境管理中是一个关键问题。然而，现有的毒性评估模型缺乏预测骨毒性的专门数据集和算法。在我们的研究中，我们收集了骨毒性分子，并使用了各种大型语言模型，包括DeepSeek和ChatGPT，以及传统的机器学习方法来预测它们的性质。其中，DeepSeek R1和ChatGPT o3模型的ACC值分别为0.87和0.88。我们的研究结果表明，机器学习方法可以帮助评估药物开发过程中有害物质对骨骼健康的影响，改进安全方案，减轻骨骼副作用，并提高治疗结果和公共安全。此外，它强调了大语言模型在预测分子毒性方面的潜力及其在健康和化学科学领域的意义。链接网址：https://pubs.acs.org/doi/10.1021/acs.jcim.5c00275 DOI：https://doi.org/10.1021/acs.jcim.5c00275 5.【配体】大多数Von Hippel-Lindau肿瘤抑制因子（VHL）的配体结合在HIF-1α结合位点。与VHL上的变构位点结合的配体在蛋白质降解领域具有很高的价值，因此，针对VHL上的Cys77进行了共价命中鉴定活动。Hit 2选择性地与VHL上的Cys77结合，而不会烷基化长素b上的活性Cys89。Hit 2表现出时间和浓度依赖性标记，其kinact/KI为0.30 M-1 s-1，并且不影响HIF-1α位点的结合。对该命中配体进行了优化，得到的化合物15具有较好的效力和标记性。确定了接近模拟物的x射线结构，揭示了化合物在VHL表面的浅槽中结合。这是第一个与VHL上的变构位点共价结合的小分子，为进一步优化提供了一个合适的起点。链接网址：https://pubs.acs.org/doi/10.1021/acsmedchemlett.4c00582 DOI：https://doi.org/10.1021/acsmedchemlett.4c00582 6.【无监督学习】许多罕见事件采样策略的主要挑战是确定捕获最慢相关运动的进度坐标。因此，能够以无监督的方式识别进度坐标的机器学习方法引起了仿真社区的极大兴趣。在这里，我们开发了一种通用方法，通过对采样构象中的异常值进行深度学习（DL），在加权集成（we）罕见事件采样过程中“实时”识别进度坐标。我们的方法识别系统采样构象的潜在空间模型中的异常值，该模型使用卷积变分自编码器进行周期性训练。作为原理证明，我们应用dl增强的we方法来模拟NTL9蛋白折叠过程。为了实现快速测试，我们的模拟使用生成的细粒度马尔可夫状态模型传播离散状态合成分子动力学轨迹。结果表明，我们的离群值动态DL在估计折叠速率常数方面的效率提高了3倍。我们的工作是在罕见事件采样中慢坐标的无监督学习方面向前迈出的重要一步。链接网址：https://pubs.acs.org/doi/10.1021/acs.jctc.4c01136 DOI：https://doi.org/10.1021/acs.jctc.4c01136 上下滚动查看更多药企动态2025年3月21日【诺华】首个！诺华「伊普可泮」获批治疗C3肾小球病2025年3月21日【恒瑞】恒瑞医药JAK1抑制剂「艾玛昔替尼」获批上市2025年3月20日【赛诺菲】赛诺菲19亿美元收购一款潜在FIC自免双抗2025年3月20日【Alnylam Pharmaceuticals】首款RNAi疗法获FDA批准治疗ATTR淀粉样变性心肌病2025年3月19日【罗氏】超10亿美元！罗氏与Oxford合作开发基于新靶点抗体新药2025年3月19日【恒瑞】恒瑞HER2 ADC治疗宫颈癌再入突破性疗法各动态具体信息，请滑动下方文字 1.【诺华】 3月21日，诺华宣布Fabhalta（iptacopan，伊普可泮）已获得FDA批准用于治疗成人C3肾小球病（C3G），以减少蛋白尿。这是第一个获批上市的C3G疗法。链接网址请戳我 2.【恒瑞】 3月21日，NMPA官网显示，恒瑞医药JAK1抑制剂艾玛昔替尼片（SHR0302）的上市申请已获批准，用于非甾体抗炎药（NSAIDs）治疗疗效欠佳或不耐受的活动性强直性脊柱炎成人患者。链接网址请戳我 3.【赛诺菲】 3月20日，赛诺菲宣布与Dren Bio达成最终协议，赛诺菲将收购DR-0201，这是一种靶向双特异性髓系细胞衔接器（MCE），在临床前和早期临床研究中已显示出强大的B细胞耗竭作用。链接网址请戳我 4.【Alnylam Pharmaceuticals】 3月20日，Alnylam Pharmaceuticals宣布其RNAi疗法Amvuttra（Vutrisiran）获批用于治疗ATTR淀粉样变性心肌病（ATTR-CM）。这是ATTR-CM领域首款RNAi疗法。链接网址请戳我 5.【罗氏】 3月19日，罗氏（Roche）宣布与英国生物技术公司Oxford BioTherapeutics达成了一项价值超过10亿美元的生物技术合作，旨在开发新的抗体癌症靶点。链接网址请戳我 6.【恒瑞】 3月19日，据CDE官网公示，恒瑞开发的HER2 ADC药物注射用SHR-A1811（瑞康曲妥珠单抗）拟被纳入突破性治疗，适应症为既往接受含铂化疗及免疫检查点抑制剂治疗失败的HER2表达（IHC≥1+）的复发或转移性宫颈癌。链接网址请戳我上下滚动查看更多会议信息 2025年3月27-28日上海恺默信息咨询有限公司举办SIT 2025第七届小分子药物创新峰会 2025年4月17-18日商图举办IGC 2025 第九届免疫基因及细胞治疗大会 2025年6月27-28日美国华人生物医药科技协会中国分会（CBA-China）主办，苏州云程万川（盛杰）承办2025美国华人生物医药科技协会CBA-China中国年会各会议具体详情和参会方式，请滑动下方文字 SIT 2025第七届小分子药物创新峰会主办方：上海恺默信息咨询有限公司会议时间：2025年3月27-28日会议地点：上海会议主旨：聚焦减重药物的突破性研究、多肽药物的最新进展以及不可成药靶点的攻克。将深入探讨小分子药物的创新路径，分享业内最前沿的技术与策略。共同探索小分子药物的无限可能，携手推动药物研发领域的持续进步。链接网址请戳我IGC 2025 第九届免疫基因及细胞治疗大会主办方：商图会议时间：2025年4月17-18日会议地点：北京会议主旨：深度解析免疫细胞治疗、干细胞与外泌体治疗、基因编辑及基因治疗、mRNA疫苗及药物、人用与兽用疫苗、免疫治疗抗体及联合治疗等领域的技术挑战与热点，促进国内产学研医的深入交流与合作。链接网址请戳我 2025美国华人生物医药科技协会CBA-China中国年会主办方：美国华人生物医药科技协会中国分会会议时间：2025年6月27-28日会议地点：苏州会议主旨：立足国际视野及产业高度，以创新为引领，围绕全球最新监管动态、新技术/AI革新及成果转化、先进治疗技术、药物创新开发、全球药物开发策略、新兴市场出海布局等前沿热门话题，共同探索前沿技术创新与全球医药合作，促进新质生产力培育及未来产业发展，推动全球健康事业新发展。链接网址请戳我上下滚动查看更多版权信息本文内容均由小编收集于公开的各个网络平台，发布的目的仅为了方便大家一站式了解AIDD行业信息，并未对发布源头进行真实性验证。如您发现相关信息有任何版权侵扰或者信息错误，请及时联系AIDD Pro（请添加微信号18515220072）进行删改处理。原创内容未经授权，禁止转载至其他平台。有问题可发邮件至yangzhuoqi@stonewise.cn 关注我，更多资讯早知道↓↓↓

疫苗临床结果临床研究紧急使用授权

2025-03-21

·Endpoints

FDA approves Novartis’ Fabhalta in another rare kidney disease

Novartis’ Fabhalta notched another FDA approval, this time in adults with an ultra-rare kidney disease called C3 glomerulopathy. Fabhalta is the first drug approved specifically to treat C3 glomerulopathy, or C3G, which is a progressive disease that damages the tiny filters in the kidney. In serious cases, the disease can eventually lead to kidney failure and lifelong dialysis. Fabhalta comes as a capsule taken twice a day. It’s also Fabhalta’s third approval — in late 2023, the drug got the green light for a rare blood disorder called paroxysmal nocturnal hemoglobinuria, and more recently it got an accelerated approval in a kidney disease called IgA nephropathy. The FDA’s decision in C3G was based on results from a Phase 3 study that showed that, alongside supportive care, treatment with Fabhalta led to a 35% reduction in proteinuria at 6 months compared to the placebo arm — meeting the primary endpoint of the study. Novartis in October also shared longer-term results that showed the proteinuria reduction was sustained through one year. In C3G, a part of the immune system known as the complement system is abnormally activated, and proteins build up in the small filters in the kidneys. Fabhalta blocks a protein that’s part of the complement system. The FDA had previously scheduled an advisory committee meeting to review Fabhalta in C3G, but canceled the meeting in February. “We had to produce, initially, six months of data, and then they [FDA] required 12 months of data. We submitted the 12 months of data, and the response was, we don’t deem it necessary anymore to have an adcomm. We don’t have more specifics than that,” Novartis’ US president Victor Bulto told Endpoints News in an interview ahead of the approval. When asked whether the cancellation was related to the new Trump administration, Bulto said that he didn’t know. “We only received a typical FDA communication saying that they didn’t deem it as necessary,” he said. Novartis has been building a suite of new kidney disease drugs, starting with Fabhalta. It’s also awaiting an FDA decision in the first half of the year on a drug called atrasentan in IgA nephropathy. And the pharma company is studying another drug called zigakibart, an anti-APRIL antibody, in Phase III for the same disease. Novartis acquired both drugs in its purchase of Chinook Therapeutics in 2023 for $3.2 billion.

临床3期临床结果上市批准并购加速审批

2025-03-21

·BioSpace

Novartis’ Fabhalta Approved as First Therapy for Rare Kidney Disease

iStock, Imagesrouges This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy. Novartis has secured a third FDA approval for Fabhalta—and the first approval for C3 glomerulopathy to reduce proteinuria, the company announced Friday. C3 glomerulopathy (C3G) is a rare kidney disease that deposits protein fragments in the glomeruli, which can progress to end-stage kidney disease within 20 years of diagnosis. The average age at diagnosis is 23 years, according to Novartis. The approval “was largely expected” after Novartis’ Phase III APPEAR-C3G study met the primary endpoint of proteinuria reduction from baseline at six months compared to placebo, William Blair analysts said in a Friday investor note. Novartis won the first FDA approval for Fabhalta in December 2023 in paroxysmal nocturnal hemoglobinuria, a rare blood disease. The company then secured accelerated approval in 2024 for primary immunoglobulin A nephropathy, another kidney disease involving protein deposits. APPEAR-C3G, a six-month double-blind study, tested a twice-daily oral formulation of Fabhalta followed by a six-month open label period where all patients got the small molecule treatment, including those in the previous period’s placebo arm. Results showed a reduction in proteinuria that Novartis said could be detected as early as 14 days after treatment and was sustained for one year on the drug. Novartis reported no new safety signals. Side effects included the common cold and viral infections. Fabhalta can cause serious infections—including Streptococcus pneumoniae , which causes pneumonia, and Neisseria meningitidis , which causes meningitis—caused by “encapsulated bacteria,” and therefore will “only be available through a Risk Evaluation and Mitigation Strategy (REMS) that requires specific vaccinations,” according to the company. “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,” Carla Nester, co-investigator of the APPEAR-C3G trial, said in a statement. The approval also marks another win for Novartis this week. On Wednesday, the company presented Phase III data at the Muscular Dystrophy Association’s Clinical & Scientific Conference showing that an intrathecal (IT) formulation of its spinal muscular atrophy gene therapy Zolgensma—approved in 2019 as an intravenous treatment for children under two years of age—appears to be effective in treating older SMA patients. The company plans to approval of the IT formulation in the first half of this year.

上市批准临床3期临床结果基因疗法

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外链

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

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适应症	国家/地区	公司	日期
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

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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
C3肾小球病	申请上市	中国	Novartis Pharma Schweiz AG	2024-09-05
C3肾小球病	申请上市	中国	Novartis Pharma Produktions GmbH	2024-09-05
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

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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


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)	齋憲壓構淵鑰築醖鬱鏇(醖網廠餘鬱願構顧積膚) = 鹹夢繭顧築獵艱鹽願築齋願選鹽選憲範鏇製鹽 (壓鹹鏇製淵襯淵選簾鏇 ) 更多	积极	2025-01-07
				Placebo (C5i-experienced patients)	齋憲壓構淵鑰築醖鬱鏇(醖網廠餘鬱願構顧積膚) = 鏇齋壓衊構鏇淵糧獵齋齋願選鹽選憲範鏇製鹽 (壓鹹鏇製淵襯淵選簾鏇 ) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Iptacopan monotherapy 200 mg twice daily	憲遞網鏇選簾糧鬱鹽鹹(襯繭廠鏇鑰顧齋觸範夢) = 襯獵鹹觸範鏇積壓遞網選廠膚淵選餘網襯糧選 (鹽簾願網築鏇範壓獵膚, 87.4) 更多	-	2024-12-09
				Eculizumab	憲遞網鏇選簾糧鬱鹽鹹(襯繭廠鏇鑰顧齋觸範夢) = 繭鬱簾顧願壓鹽夢壓壓選廠膚淵選餘網襯糧選 (鹽簾願網築鏇範壓獵膚, 77.2) 更多
ASH2024	N/A	冷凝集素病	-	Iptacopan 200 mg	糧窪襯製觸齋壓鏇醖壓(餘壓願蓋鹽憲顧膚簾糧) = 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. 築鹹齋餘鬱選窪糧遞簾 (淵糧夢壓淵選願窪獵糧 )	-	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	網廠築遞廠網積網糧網(遞艱鑰網蓋鑰築鹽淵顧) = 製獵選餘醖餘獵構膚觸願築鹹繭窪構範艱顧淵 (獵範憲醖觸鹽淵鬱網窪, 36 ~ 51) 更多	积极	2024-08-07
				Placebo	網廠築遞廠網積網糧網(遞艱鑰網蓋鑰築鹽淵顧) = 襯獵鏇醖鬱夢鹹糧簾鹹願築鹹繭窪構範艱顧淵 (獵範憲醖觸鹽淵鬱網窪, -5 ~ 21) 更多
NCT04154787 (CTgov)	临床2期	特发性膜性肾病	37	Rituximab	襯簾觸觸糧範餘選蓋繭(膚廠艱積窪遞窪餘淵顧) = 餘夢夢鬱鑰鹹繭築簾鹽襯鏇衊艱願艱遞齋繭窪 (憲築鑰選壓選襯窪糧窪, 顧遞鬱顧選鑰鹽醖願艱 ~ 衊糧鬱淵製鑰觸餘遞醖) 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)	願衊憲選鹹憲膚製願範(壓夢艱鏇構鏇蓋艱糧鑰) = 醖窪蓋築齋範願糧淵膚膚襯醖齋醖築衊壓糧願 (簾壓膚鹽廠鹽遞膚艱廠 )	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	網網窪鹹築遞餘廠蓋膚(獵壓獵顧遞簾觸構蓋願) = 鹹範壓願膚齋網壓蓋網鑰艱鑰衊醖鏇製簾積蓋 (艱蓋選鑰簾願範製製鏇 ) 更多	积极	2024-05-14
				Pegcetacoplan	網網窪鹹築遞餘廠蓋膚(獵壓獵顧遞簾觸構蓋願) = 積鏇糧壓獵膚窪襯憲蓋鑰艱鑰衊醖鏇製簾積蓋 (艱蓋選鑰簾願範製製鏇 )
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	鬱膚窪積鏇製壓鑰淵憲(膚構觸構獵蓋選廠鏇願) = 壓窪醖製選窪築糧網鑰積鏇顧齋觸選餘鏇選廠 (憲選鬱積製選鹽網繭憲 ) 更多	积极	2024-05-14
NCT04747613 (APPOINT-PNH \| APPLY-PNH \| roll-over extension, EHA2024)	临床3期	阵发性睡眠性血红蛋白尿症	61	Iptacopan	壓築簾壓餘積壓繭壓簾(醖艱鏇簾願觸選餘艱網) = 製憲遞憲齋醖鬱艱襯築窪鬱壓餘蓋鏇簾構築鏇 (糧繭遞簾糧顧顧繭積顧 )	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	餘鏇襯餘網壓獵艱鬱窪(鹽壓糧網憲願網襯獵衊) = 構觸構繭鹹網積遞糧選網製範壓憲遞餘積醖觸 (製廠窪製鬱製糧鬱壓蓋, -16.1%) 更多	积极	2024-05-14
				Anti-C5 inhibitors	餘鏇襯餘網壓獵艱鬱窪(鹽壓糧網憲願網襯獵衊) = 壓艱構遞積構遞鑰淵夢網製範壓憲遞餘積醖觸 (製廠窪製鬱製糧鬱壓蓋, -16.9%) 更多