A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)

The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to <18 years.

开始日期2025-12-08

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

NCT06934967

/ Not yet recruiting临床3期

An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to < 18 years of age.

开始日期2025-07-28

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

NCT06931691

/ Not yet recruitingN/A

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China

The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.

开始日期2025-04-30

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

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）

2025-06-03·PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Host complement C3 promotes malaria transmission by killing symbiotic bacteria in the mosquito midgut

Article

作者: Li, Meilin ; Zhou, Taoli ; Huang, Wei ; Liu, Taiping ; Jiang, Lubin ; Tan, Nie ; Gao, Yuanli ; Xu, Wenyue ; Li, Jian ; Guo, Shuai ; Jiao, Shiming ; Jiao, Zhiwei ; Zhang, Jian ; Lin, Zurui ; Li, Jianyong ; He, Biao ; Fan, Yongling ; Zhu, Feng ; Wang, Sibao

Host-derived factors ingested during mosquito blood feeding are poorly understood modulators of malaria transmission. Here, we demonstrated that host complement C3, acquired by mosquitoes during Plasmodium infection, significantly enhanced rodent malaria infection in laboratory-reared mosquitoes. This effect was recapitulated in field-caught Anopheles sinensis mosquitoes, confirming its relevance to malaria transmission in a more natural setting. Moreover, host-derived C3 significantly reduced the efficacy of anti-Pfs25 antibodies in blocking malaria transmission. Mechanistically, host-derived C3 lyses the mosquito midgut symbiont Elizabethkingia anophelis ( E. anophelis )—a bacterium that intrinsically suppresses parasite development by blocking the zygote-to-ookinete transition. Strikingly, host-derived C3 in mosquitoes appears to be activated by the alternative pathway, and inhibiting Factor B with Iptacopan (LNP023) reduced Plasmodium falciparum ( P. falciparum ) infection, while increased the efficacy of anti-Pfs25 antibodies to blocking P. falciparum transmission in the standard membrane-feeding assay. Therefore, this study describes a strategy of the malaria parasite to utilize host complement C3 to promote its transmission and provides us with an avenue to block malaria transmission and improve the blocking efficacy of anti-Pfs25 antibodies by the inhibition of C3 activation.

2025-06-01·EJHaem

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

Article

作者: Fermont, Jilles M. ; Brindel, Isabelle ; Kelly, Richard J. ; Holt, Matthew ; de Fontbrune, Flore Sicre ; Dahlke, Marion ; Maafa, Lynda ; Ansari, Soudeh ; de Latour, Régis Peffault

ABSTRACT:

Background:

Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive patients demonstrated significant haemoglobin concentration improvements.

Methods:

We used target trial emulation to retrospectively predict outcomes if APPOINT‐PNH trial patients had received C5 inhibitors instead of iptacopan. Estimates were derived from the real‐world APPEX cohort treated with routine C5 inhibitors. The study used benchmarking and comparative effectiveness to evaluate the haematological response in APPOINT‐PNH if patients had received C5 inhibitors. Treatment effect was estimated using propensity scores to model the probability of trial inclusion based on baseline covariates, followed by fitting an outcome model to the APPEX cohort.

Results:

The analysis of 125 patients showed all estimated treatment effects (95% confidence interval) favoured iptacopan over C5 inhibitors: differences in the proportion of patients achieving haemoglobin increase from baseline of ≥ 2 g/dL, 68.2% (40.9–95.6); haemoglobin levels of ≥ 12 g/dL, 53.4% (31.4–75.3); transfusion independence, 38.8% (15.1–62.5); ratio of percent change from baseline in lactate dehydrogenase levels, 0.51 (0.40–0.67); change from baseline in reticulocytes, −75.5 × 109/L (−106.9, −44.2).

Conclusions:

Results indicate C5 inhibitor‐naive patients with PNH may experience greater haematological response with iptacopan than with C5 inhibitors.

Trial Registration:

ClinicalTrials.gov identifier: NCT05842486

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.

470

项与盐酸伊普可泮相关的新闻（医药）

2025-06-13

·诺华集团

诺华飞赫达®在阵发性睡眠性血红蛋白尿症新患者群体中显示出具有统计学显著且临床意义的血红蛋白改善

在ⅢB期APPULSE-PNH研究中，转换为飞赫达®治疗的阵发性睡眠性血红蛋白尿症（PNH）成人患者的血红蛋白（Hb）水平平均升高2.01g/dL，显示出具有临床意义的显著改善1,2。APPULSE-PNH研究评估了飞赫达®在基线血红蛋白（Hb）水平高于关键Ⅲ期研究人群中的疗效，拓宽了临床证据基础1,2。飞赫达®治疗期间无患者需要输血、发生突破性溶血（BTH）或任何主要不良血管事件1。接受飞赫达®治疗的患者在研究第84天和第168天时，疲劳症状评分较基线平均改善4.88分和4.29分（通过FACIT-疲劳评分测量）1。诺华宣布了APPULSE-PNH ⅢB期研究的积极结果：该研究评估了每日两次口服单药飞赫达®（伊普可泮）在基线血红蛋白水平大于等于10g/dL(Hb≥10g/dL)、且从抗补体C5治疗（依库珠单抗和瑞利珠单抗）转换而来的PNH成人患者中的疗效与安全性1。接受飞赫达®治疗24周后，Hb水平平均提升2.01g/dL（95%CI：1.74，2.29），大多数患者达到正常或接近正常水平1，数据将于2025年欧洲血液学会（EHA）年会公布。伦敦国王学院医院及伦敦国王学院血液科顾问医师Austin Kulasekararaj当前，仍存在部分PNH患者的治疗需求未被依库珠单抗和瑞利珠单抗满足，APPULSE-PNH研究的积极结果进一步证实，对于基线Hb水平高于既往研究入组患者的群体，飞赫达®可实现具有临床意义的血红蛋白改善，同时为患者提供口服单药的治疗选择。研究期间无患者需要输血，绝大多数患者（92.7%）Hb水平超过12g/dL，即正常或接近正常水平1。接受飞赫达®治疗的患者在第168天时报告了具有临床意义的疲劳改善（通过FACIT-疲劳评分测量），其绝对值水平与普通人群报告的水平相当1,3,4。此外，接受飞赫达®治疗的患者维持了对血管内溶血的控制并解决了血管外溶血的情况，表现为乳酸脱氢酶水平降低（<1.5倍正常上限）和网织红细胞绝对值下降1。诺华公司致力于推动能改变诊疗实践并显著改善PNH患者及其照护者生活的研究与创新。APPULSE-PNH的新数据，结合APPLY-PNH和APPOINT-PNH研究Ⅲ期延长试验的随访结果，进一步证实了飞赫达®的有效性和安全性，患者可实际获益。飞赫达®是目前首个也是唯一可用于治疗PNH成人的口服单药，且不受既往治疗经验限制。除APPULSE-PNH研究外，最初纳入APPLY-PNH和APPOINT-PNH Ⅲ期研究的患者的更长随访数据也将在EHA年会上公布1。在APPULSE-PNH研究、APPLY-PNH和APPOINT-PNH Ⅲ期研究的延长随访数据中，飞赫达®耐受性良好，未发现新的安全信号，与既往报告数据一致1,5。↑滑动查看更多↑关于阵发性睡眠性血红蛋白尿症（PNH）PNH是一种罕见、慢性且严重的补体介导的血液疾病6。PNH患者的部分造血干细胞（位于骨髓中，可生长发育为红细胞、白细胞和血小板）发生获得性突变，导致其产生易被补体系统过早破坏的红细胞6,7。从而引发血管内溶血（红细胞在血管内被破坏）和血管外溶血（红细胞主要在脾脏和肝脏中被破坏），表现为贫血（循环红细胞水平较低）、血栓形成（形成血凝块）、疲劳和其他衰弱症状6,7。据估计，全球每百万人中约有10-20人患有PNH6。尽管PNH可在任何年龄发生，但常见于30-40岁的人群8,9。PNH仍存在大量需求仍未被抗补体C5治疗（依库珠单抗或瑞利珠单抗）满足。抗补体C5治疗（依库珠单抗或瑞利珠单抗）通常每2周至8周需接受一次静脉输注，治疗访视（包括路程、等待、输注和恢复时间）大约需要4-5小时10。即使接受抗补体C5治疗，大部分PNH患者仍存在贫血，还有部分患者依赖输血7,11-16。关于APPULSE-PNHAPPULSE-PNH（NCT05630001）是一项Ⅲb期、多国、多中心、单臂、开放标签研究，旨在评估从抗补体C5治疗（依库珠单抗或瑞利珠单抗）转换为每日两次口服200mg飞赫达®（伊普可泮）单药治疗的成年PNH患者的疗效和安全性，该试验共纳入了52例患者，接受24周的飞赫达®治疗2。入组的受试者需要在筛选前接受稳定的抗补体C5治疗（依库珠单抗或瑞利珠单抗）至少6个月，且平均血红蛋白（Hb）水平≥10g/dL，并且在此期间未输注红细胞2,17。主要终点为飞赫达®治疗24周后Hb水平较基线的变化2,17。关于APPLY-PNHAPPLY-PNH（NCT04558918）是一项Ⅲ期、随机、多国、多中心、标准治疗对照、开放标签试验。通过评估在随机分组前6个月内接受稳定的抗C5治疗方案但仍存在残留贫血（Hb<10g/dL）的成人患者，转换为飞赫达®治疗是否优于继续抗C5治疗（依库珠单抗或瑞利珠单抗），评价每日两次口服飞赫达®（伊普可泮）单药（200mg）治疗PNH的疗效和安全性18,19。关于APPOINT-PNH APPOINT-PNH（NCT04820530）是一项III期、多国、多中心、开放标签、非对照单臂试验，旨在评估每日两次口服单药飞赫达®（伊普可泮）200mg治疗在未接受过补体抑制剂治疗，包括抗补体C5治疗（依库珠单抗或瑞利珠单抗），的成年PNH患者中的有效性和安全性20,21。关于Fabhalta®/飞赫达®（伊普可泮）Fabhalta®/飞赫达®（伊普可泮）是一种口服的、补体旁路途径的B因子抑制剂22,23。诺华公司研发的Fabhalta®分别于2023年12月和2024年5月获得美国食品药品监督管理局（FDA）和欧盟委员会（EC）批准用于治疗成人PNH；并于2024年8月，在美国获得加速批准，用于降低有快速进展风险的成人原发性IgA肾病（IgAN）患者的蛋白尿（通常UPCR≥1.5g/g）1,24,25。2025年，Fabhalta®获FDA和EC批准用于治疗成人C3肾小球病（C3G），减少蛋白尿，成为目前首个且唯一一款用于治疗该疾病的获批疗法26-32。Fabhalta®/飞赫达®正在多种罕见肾病中开展研究，包括非典型溶血性尿毒症综合征（aHUS）、免疫复合物膜增生性肾小球肾炎（IC-MPGN）和狼疮性肾炎（LN）。正在开展的研究将评估这些研究性适应症的安全性和疗效特征，以支持潜在的监管申报33-36。— 免责声明 — （滑动查看更多）1. 本资料目的在于传递医药前沿信息和研究进展，非广告用途。2. 本资料中涉及的信息仅供参考，请遵从医生或其他医疗专业人士的意见或指导。本新闻稿包含1995年《美国私人证券诉讼改革法》意义上的前瞻性陈述。前瞻性陈述通常可通过“潜在”、“可能”、“意愿”、“计划”、“可能”、“可能”、“预期”、“研究”、“驱动”、“仍然”、“正在进行”、“探索”、“目标”、“预期”、“估计”或类似术语等词语识别，或通过对此产品的潜在上市批准、新适应症或标签的明示或暗示讨论，或关于此类产品的潜在未来收入识别。你不应该过分依赖这些说法。此类前瞻性声明基于我们目前对未来事件的信念和期望，并受到显著的已知和未知风险和不确定性的影响。如果这些风险或不确定性中的一个或多个具体化，或如果基础假设证明不正确，实际结果可能与前瞻性声明中的结果存在实质性差异。不能保证此产品将在任何市场或任何特定时间提交或批准销售或用于任何其他适应症或标签。也不能保证此类产品在未来将取得商业成功。尤其是，我们对此产品的期望可能会受到以下因素的影响：研究和开发中固有的不确定性，包括临床试验结果和现有临床数据的额外分析；监管行动或一般延迟或政府监管；医疗保健成本控制的全球趋势，包括政府、付款人和一般公共定价以及提高定价透明度的压力和要求；我们获得或维持专有知识产权保护的能力；医生和患者的特殊处方偏好；一般政治、经济和商业条件，包括减轻大流行性疾病的影响和努力；安全性、质量、数据完整性或制造问题；潜在或实际的安全数据和数据隐私违反，或我们的信息技术系统的破坏，以及诺华向美国证券交易委员会备案的当前表格20-F中提及的其他风险和因素。诺华公司在本新闻稿中提供截至本日期的信息，并且没有义务因新信息、未来事件或其他原因更新本新闻稿中包含的任何前瞻性声明。— 参考文献 — （滑动查看更多）1.Novartis.Dataonfile.2.Clinicaltrials.gov.NCT05630001.SingleArm,OpenLabelTrialWithIptacopanTreatmentfor24Weeks,inPatientsonStableRegimenofAnti-C5WhoSwitchtoIptacopan.(APPULSE).Availablefrom:https://clinicaltrials.gov/study/NCT05630001AccessedMay,2025.3.MontanI,LöweB,CellaD,MehnertA,HinzA.GeneralPopulationNormsfortheFunctionalAssessmentofChronicIllnessTherapy(FACIT)-FatigueScale.ValueHealth.2018;21(11):1313-1321.doi:10.1016/j.jval.2018.03.0134.CellaD,LaiJS,ChangCH,PetermanA,SlavinM.FatigueincancerpatientscomparedwithfatigueinthegeneralUnitedStatespopulation.Cancer.2002;94(2):528-538.doi:10.1002/cncr.102455.DighririIM,Al-QahtaniRM,AlmutairiAO,etal.IptacopanEfficacyandSafetytoTreatParoxysmalNocturnalHemoglobinuria(PNH):ASystematicReviewandMeta-Analysis.Cureus.2024;16(8):e67830.6.CançadoRD,AraújoADS,SandesAF,etal.Consensusstatementfordiagnosisandtreatmentofparoxysmalnocturnalhaemoglobinuria.HematolTransfusCellTher.2021;43(3):341-348.7.DingliD,MatosJE,LehrhauptK,etal.TheburdenofillnessinpatientswithparoxysmalnocturnalhemoglobinuriareceivingtreatmentwiththeC5-inhibitorseculizumaborravulizumab:resultsfromaUSpatientsurvey.AnnHematol.2022;101(2):251-263.8.HillA,DeZernAE,KinoshitaT,BrodskyRA.Paroxysmalnocturnalhaemoglobinuria. NatRevDisPrimers.2017;3:17028.9.RöthA,MaciejewskiJ,NishimuraJI,JainD,WeitzJI.Screeninganddiagnosticclinicalalgorithmforparoxysmalnocturnalhemoglobinuria:Expertconsensus. EurJHaematol.2018;101(1):3-11.10.LevyAR,DysartL,PatelY,etal.ComparisonofLostProductivityDuetoEculizumabandRavulizumabTreatmentsforParoxysmalNocturnalHemoglobinuriainFrance,Germany,Italy,Russia,Spain,theUnitedKingdom,andtheUnitedStates.Blood.2019;134(Supplement_1):4803.11.McKinleyCE,RichardsSJ,MunirT,etal.ExtravascularHemolysisDuetoC3-LoadinginPatientswithPNHTreatedwithEculizumab:DefiningtheClinicalSyndrome.Blood.2017;130(Supplement1):3471.12.RisitanoAM,MarottaS,RicciP,etal.Anti-complementTreatmentforParoxysmalNocturnalHemoglobinuria:TimeforProximalComplementInhibition?APositionPaperFromtheSAAWPoftheEBMT.FrontImmunol.2019;10:1157.13.ShammoJ,KimJ,GeorgetM,etal.P796:Hospitalizationinpatientswithparoxysmalnocturnalhemoglobinuria:aretrospectiveanalysisofobservationalstudydatafromtheUnitedStates.Hemasphere.2023;7(Suppl):e22585a2.14.DebureauxPE,KulasekararajAG,CacaceF,etal.Categorizinghematologicalresponsetoeculizumabinparoxysmalnocturnalhemoglobinuria:amulticenterreal-lifestudy.BoneMarrowTransplant.2021;56(10):2600-2602.15.SchrezenmeierH,KulasekararajA,MitchellL,etal.One-yearefficacyandsafetyofravulizumabinadultswithparoxysmalnocturnalhemoglobinurianaïvetocomplementinhibitortherapy:open-labelextensionofarandomizedstudy.TherAdvHematol.2020;11:2040620720966137.16.YoungNS,MeyersG,SchrezenmeierH,HillmenP,HillA.Themanagementofparoxysmalnocturnalhemoglobinuria:recentadvancesindiagnosisandtreatmentandnewhopeforpatients.SeminHematol.2009;46(1Suppl1):S1-S16.17.RisitanoAM,AratenDJ,KuterD,etal.Pb2063:APPULSE-PNH:AphaseIIIBtrialtoevaluatetheefficacyandsafetyofswitchingtoiptacopaninpatientswithparoxysmalnocturnalhemoglobinuria(PNH)onanti-c5therapywithhemoglobin>10g/dl.Hemasphere.2023;7(Suppl):e08587b7.18.PeffaultdeLatourR,RöthA,KulasekararajAG,etal.OralIptacopanMonotherapyinParoxysmalNocturnalHemoglobinuria.NEnglJMed.2024;390(11):994-1008.doi:10.1056/NEJMoa230869519.Clinicaltrials.gov.NCT04558918.StudyofEfficacyandSafetyofTwiceDailyOralLNP023inAdultPNHPatientsWithResidualAnemiaDespiteAnti-C5AntibodyTreatment(APPLY-PNH).Availablefrom:https://clinicaltrials.gov/study/NCT04558918AccessedMay,2025.20.RisitanoAM,HanB,UedaY,etal.OralComplementFactorBInhibitorIptacopanMonotherapyImprovesHemoglobintoNormal/Near-NormalLevelsinParoxysmalNocturnalHemoglobinuriaPatientsNaïvetoComplementInhibitors:PhaseIIIAPPOINT-PNHTrial.Presentedat:49thAnnualMeetingoftheEuropeanSocietyforBloodandMarrowTransplantation(EBMT);April23-36,2023;Paris,France.21.Clinicaltrials.gov.NCT04820530.StudyofEfficacyandSafetyofTwiceDailyOralIptacopan(LNP023)inAdultPNHPatientsWhoAreNaivetoComplementInhibitorTherapy(APPOINT-PNH).Availablefrom:https://clinicaltrials.gov/study/NCT04820530AccessedMay,2025.22.KavanaghD,BombackA,VivarelliM,etal.EfficacyandSafetyofIptacopaninPatientswithC3Glomerulopathy:ResultsfromthePhase3APPEAR-C3GTrial.PresentedatEuropeanRenalAssociation(ERA)Congress;May25,2024;Stockholm,Sweden.23.SmithRJ,KavanaghD,VivarelliM,etal.EfficacyandsafetyofiptacopaninpatientswithC3glomerulopathy:12-MonthresultsfromthePhase3APPEAR-C3Gstudy.PresentedatAmericanSocietyofNephrology(ASN)KidneyWeek2024;October23-27,2024;SanDiego,CA.24.Novartis.Pressrelease.NovartisreceivesFDAapprovalfor伊普可泮(iptacopan),offeringsuperiorhemoglobinimprovementintheabsenceoftransfusionsasthefirstoralmonotherapyforadultswithPNH.Availablefrom:https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-伊普可泮-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnhAccessedMay,2025.25.Novartis.Pressrelease.NovartisreceivesFDAacceleratedapprovalfor伊普可泮(iptacopan),thefirstandonlycomplementinhibitorforthereductionofproteinuriainprimaryIgAnephropathy(IgAN).Availablefrom:https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-伊普可泮-iptacopan-first-and-only-complement-inhibitor-reduction-proteinuria-primary-iga-nephropathy-iganAccessedMay,2025.26.Novartis.Pressrelease.NovartisreceivesthirdFDAapprovalfororal伊普可泮(iptacopan)–thefirstandonlytreatmentapprovedinC3glomerulopathy(C3G).Availablefrom:https://www.novartis.com/news/media-releases/novartis-receives-third-fda-approval-oral-伊普可泮-iptacopan-first-and-only-treatment-approved-c3-glomerulopathy-c3gAccessedMay,2025.27.Novartis.Pressrelease.Novartisoral伊普可泮(iptacopan)receivespositiveCHMPopinionforthetreatmentofadultslivingwithC3glomerulopathy(C3G).Availablefrom:https://www.novartis.com/news/media-releases/novartis-oral-伊普可泮-iptacopan-receives-positive-chmp-opinion-treatment-adults-living-c3-glomerulopathy-c3gAccessedMay,2025.28.伊普可泮.USFDAPrescribinginformation.EastHanover,NJ:NovartisPharmaceuticalsCorp;2024.Availablefrom: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218276s001lbl.pdfAccessedMay,2025.29.伊普可泮.EMASummaryofProductCharacteristics.NovartisEuropharmLimited;2024.Availablefrom:https://www.ema.europa.eu/en/documents/product-information/伊普可泮-epar-product-information_en.pdfAccessedMay,2025.30.MartínB,SmithRJH.C3Glomerulopathy.In:AdamMP,FeldmanJ,MirzaaGM,etal,eds.GeneReviews®[Internet].Seattle,WA:UniversityofWashington,Seattle;1993-2025. UpdatedApril5,2018.Availablefrom:https://www.ncbi.nlm.nih.gov/books/NBK1425/AccessedMay,2025. 31.SchenaFP,EspositoP,RossiniM.ANarrativeReviewonC3Glomerulopathy:ARareRenalDisease.IntJMolSci.2020;21(2):525. 32.Caravaca-FontánF,LucientesL,CaveroT,PragaM.UpdateonC3Glomerulopathy:AComplement-MediatedDisease.Nephron.2020;144(6):272-280. 33.Clinicaltrials.gov.NCT04578834.StudyofEfficacyandSafetyofLNP023inPrimaryIgANephropathyPatients(APPLAUSE-IgAN).Availablefrom:https://clinicaltrials.gov/study/NCT04578834AccessedMay,2025.34.Clinicaltrials.gov.NCT04889430.EfficacyandSafetyofIptacopan(LNP023)inAdultPatientsWithAtypicalHemolyticUremicSyndromeNaivetoComplementInhibitorTherapy(APPELHUS).Availablefrom:https://clinicaltrials.gov/study/NCT04889430AccessedMay,2025.35.Clinicaltrials.gov.NCT05755386.StudyofEfficacyandSafetyofIptacopaninParticipantsWithIC-MPGN(APPARENT).Availablefrom:https://clinicaltrials.gov/study/NCT05755386AccessedMay,2025.36.Clinicaltrials.gov.NCT05268289.StudyofEfficacyandSafetyofLNP023inParticipantsWithActiveLupusNephritisClassIII-IV,+/-V.Availablefrom:https://clinicaltrials.gov/study/NCT05268289AccessedMay,2025.

临床3期临床结果临床成功

2025-06-12

·医脉通

胡豫教授：医疗创新驱动中国罕见血液病发展，PNH领域快速突破提供重要经验

世界卫生组织（WHO）将患病人数占总人口0.65‰-1‰的疾病或病变定义为罕见病。罕见病的发病率虽然低，但中国人口基数大，这意味着相当数量的患者身处疾病困境。罕见病长期缺乏有效治疗药物，患者平均确诊时间长达5年，需要经历7.5位医师的就诊过程1。罕见病不仅是医学难题，更需要整个医疗体系共同努力。过去十年间，中国罕见病防治事业取得了跨越式发展，药物可及性不断提升。其中，罕见血液病阵发性睡眠性血红蛋白尿症（PNH）的诊疗突破具有典型代表意义。近日，随着口服补体B因子抑制剂伊普可泮PNH全线适应症的获批，我国PNH治疗领域迈入崭新阶段。基于此，医脉通特邀请华中科技大学同济医学院附属协和医院胡豫教授围绕罕见病的防治进展、PNH的诊疗突破展开深度解读。罕见病防治工作面临诸多挑战，亟需提升对罕见病临床与科研的关注《中国罕见病定义研究报告2021》将我国罕见病定义为“新生儿发病率低于1/10000，患病率低于1/10000，患病人数低于14万的疾病（符合其一即可认定）”2。目前，全球罕见病已超过7000种，只有约5%的罕见病拥有针对性的治疗方案，我国约有2000多万罕见病患者1,3。罕见病种类繁多、发病机制复杂、诊疗难度大，给患者及其家庭带来了沉重的负担。同时，罕见病药物的研发成本高、周期长，药物可及性和可负担性仍然是我们需要努力解决的问题。可以说，罕见病防治工作面临着诸多挑战，提升罕见病关注度，已然成为完善公共卫生服务架构、推动医学科研进步、增进社会福祉的关键议题。罕见病目录发布为罕见血液病临床与科研发展指引方向近年来，国家相关部门在罕见病诊疗体系、药物研发、保障等层面，持续出台一系列支持政策，涵盖指引性纲领、罕见病药物审批、罕见病药物临床研究指导原则等。2018年，《第一批罕见病目录》的发布，121种疾病被纳入目录。2023年，《第二批罕见病目录》新增86种罕见病2。其中，首批目录中血液罕见病占比超10%，第二批目录新增病种里，血液系统罕见病比例进一步提升（表1）。罕见病目录的发布，为罕见血液病的发展指明了清晰方向。表1 罕见病目录中的血液系统疾病医疗创新驱动PNH领域快速突破，为中国罕见血液病发展提供经验PNH是一种后天获得性溶血性疾病，我国年发病率约为2.85/100万人4，已被纳入第一批罕见病目录。考虑到我国庞大的人口基数，实际PNH患病人群规模不可忽视。PNH临床主要表现为血管内溶血、骨髓衰竭和高风险并发血栓等，严重可危及生命。77%的患者在20~40岁发病4，正值人生黄金期，面临生育、职业等多重社会角色中断，叠加疾病本身的反复贫血、感染和血栓风险，生存质量与家庭经济负担均处于重压状态。有研究表明，在缺乏有效治疗药物的情况下，PNH患者的5年死亡率约为35%5。近年来，中国血液学工作者在PNH领域的深耕实践，积极参与全球多中心临床研究，系统整合国内外最新研究证据，牵头制定多项指南与共识，成为全球PNH学科发展的重要推动力。在临床和科研工作者的共同努力下，PNH诊断和治疗手段的突破改变了临床格局。流式细胞术快速发展，已成为PNH诊断的金标准。末端补体抑制剂的上市推动治疗目标从传统治疗时代的“缓解疾病症状”迈向“控制血管内溶血”，大大提高了PNH患者的生存率。新型近端补体抑制剂伊普可泮在中国快速获批，目前已覆盖既往未接受补体抑制剂治疗和接受过补体抑制剂治疗的PNH患者，进一步推动PNH治疗迈向追求“完全缓解”的更高治疗目标，帮助PNH患者重回正常工作与生活，成为社会发展的重要力量。可以说，中国PNH领域的快速发展与突破，为其他罕见血液病的发展提供了经验与参考。专家点评胡豫教授近年来，医疗创新驱动中国罕见血液病领域不断发展，取得了诸多成就。以PNH为例，流式细胞术的技术发展与标准化应用提升了检测精准度，降低疾病误诊漏诊率，缩短PNH患者的诊断周期。治疗手段方面，创新药物在国内的快速上市，推动PNH实现了从传统支持治疗到末端补体抑制剂治疗，再到新型近端补体抑制剂伊普可泮的快速跨越，推动PNH治疗目标向实现“完全缓解”大步迈进。得益于此，中国PNH患者有望摆脱疾病束缚，重新回归正常工作与生活。路漫漫其修远兮，吾将上下而求索。在攻克罕见血液病这场关乎千万人生命健康的征程中，正如PNH领域的创新与突破，中国血液临床与科研工作者不断努力，以医疗创新驱动中国罕见血液病领域不断发展！专家简介胡豫教授华中科技大学同济医学院附属协和医院华中科技大学血液病学研究所所长教育部生物靶向治疗重点实验室主任中华医学会血液学分会主委中华医学会内科学分会常委中国医师协会血液科医师分会副会长中国医学科学院学术咨询委员会学部委员国际血栓与止血学会教育委员会委员中华血液学杂志主编临床内科杂志/临床血液学杂志/临床急诊杂志主编Thrombosis Research/Thrombosis and Haemostasis 副主编国家杰青、长江特聘全国教书育人楷模全国优秀医院院长国家科技进步二等奖全国创新争先奖章何梁何利基金奖以第一或通讯作者在Cell、NEJM、JAMA、Lancet Oncology等期刊发表论文318篇，SCI 论文 231篇，其中 22 篇IF＞20，SCI 他引 2万余次，连续多年入选全球高被引学者参考文献：1.顾洁,黄延焱.罕见病诊治及全过程管理:全科医师的作用不可或缺[J].中国全科医学:0-null[2025-05-29].2.国家药品监督管理局药品审评中心. 罕见病基因治疗产品临床试验技术指导原则(试行).3.张连雪,刘远立.中国罕见病多重保障机制的现状与对策[J].罕见病研究, 2025, 4(1):7-13.4.国家卫生健康委员会. 罕见病诊疗指南（2019 年版）.5.Sharma VR. Paroxysmal nocturnal hemoglobinuria: pathogenesis, testing, and diagnosis. Clin Adv Hematol Oncol. 2013 Sep;11 Suppl 13(9):2-8. 审批码FAP0048243-102399，有效期为2025-06-11至2026-06-10，资料过期，视同作废撰写：Vera审校：Vera排版：Atai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

2025-06-12

·PipelineReview

Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH

BASEL, Switzerland I June 12, 2025 I Novartis announced positive results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta ® (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with Hb levels ≥10g/dL who switched from anti-C5 therapies (eculizumab or ravulizumab) 1 . After 24 weeks of treatment with Fabhalta, the Hb level improved on average by 2.01 g/dL (95% CI, 1.74, 2.29) with most patients achieving normal or near-normal levels 1 . Data will be presented at the European Hematology Association (EHA) Congress 2025. “Today, some patients living with PNH have unmet needs not addressed by eculizumab or ravulizumab,” said Austin Kulasekararaj, Consultant Hematologist, Kings College Hospital and Kings College London. “The positive results from APPULSE-PNH reinforce that Fabhalta can provide clinically meaningful improvements in hemoglobin among patients with higher baseline hemoglobin levels than those enrolled in previous trials, while offering an oral monotherapy for patients.” No patients required transfusion during the study, and the vast majority (92.7%) achieved Hb ≥12g/dL, reaching normal or near-normal levels 1 . Patients treated with Fabhalta also reported clinically meaningful improvements in fatigue (as measured by FACIT-Fatigue score) through Day 168, reaching absolute levels similar to those reported in the general population 1,3,4 . Furthermore, patients treated with Fabhalta maintained intravascular hemolysis control and resolved extravascular hemolysis control, as demonstrated by lactate dehydrogenase levels (<1.5 upper limit of normal) and a reduction in absolute reticulocyte count 1 . “Novartis is dedicated to advancing research and innovation that can transform care and significantly improve the lives of people living with PNH and those who support them,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “New data from APPULSE-PNH, combined with findings from the Phase III roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients. Fabhalta is the first and only oral monotherapy currently available for the treatment of adults with PNH, regardless of previous treatment experience.” Alongside APPULSE-PNH, longer-term data from patients initially included in the APPLY-PNH and APPOINT-PNH Phase III studies will be presented at EHA 1 . In APPULSE-PNH and the Phase III roll-over extension study of the APPLY-PNH and APPOINT-PNH studies, Fabhalta was well-tolerated with no new safety signals, consistent with previously reported data 1,5 . About paroxysmal nocturnal hemoglobinuria (PNH) PNH is a rare, chronic and serious complement-mediated blood disorder 6 . People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into red blood cells [RBCs], white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system 6,7 . This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots), fatigue and other debilitating symptoms 6,7 . It is estimated that approximately 10-20 people per million worldwide live with PNH 6 . Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old 8,9 . PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab). Anti-C5 therapies (eculizumab or ravulizumab) are commonly administered every 2-8 weeks as intravenous infusions, and treatment visits (including journey, waiting, infusion and recovery time) can take approximately 4 to 5 hours 10 . Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions 7,11-16 . About APPULSE-PNH APPULSE-PNH (NCT05630001) is a Phase IIIB, multinational, multicenter, single-arm, open-label study to evaluate the efficacy and safety of twice-daily oral Fabhalta ® (iptacopan) monotherapy (200mg) in adults with PNH who were switched from anti-C5 therapies (eculizumab or ravulizumab) 2 . The trial enrolled 52 participants who received Fabhalta for 24 weeks 2 . Participants enrolled were required to be on a stable regimen with anti-C5 therapies (eculizumab or ravulizumab) for at least 6 months prior to screening with average hemoglobin (Hb) ≥10g/dL and no red blood cell transfusions in this period 2,17 . The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta 2,17 . About APPLY-PNH APPLY-PNH (NCT04558918) is a Phase III, randomized, multinational, multicenter, active-comparator controlled, open-label trial to evaluate the efficacy and safety of twice-daily, oral Fabhalta ® (iptacopan) monotherapy (200mg) for the treatment of PNH by demonstrating the superiority of Fabhalta compared to anti-C5 therapies (eculizumab or ravulizumab) in adult patients presenting with residual anemia (Hb <10 g/dl) despite a stable regimen of anti-C5 treatment in the last six months prior to randomization 5,18 . About APPOINT-PNH APPOINT-PNH (NCT04820530) is a Phase III, multinational, multicenter, open-label, single-arm study to evaluate the efficacy and safety of twice-daily, oral Fabhalta ® (iptacopan) monotherapy (200mg) in adult PNH patients who are naïve to complement inhibitor therapy, including anti-C5 therapies (eculizumab or ravulizumab) 19,20 . About Fabhalta® (iptacopan) Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway 21,22 . Discovered at Novartis, Fabhalta received US Food and Drug Administration (FDA) and European Commission (EC) approval in December 2023 and May 2024 respectively for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and accelerated approval in the US in August 2024 for the reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression (generally UPCR ≥1.5 g/g 1.5 g/g) 1,23,24 . In 2025, Fabhalta received FDA and EC approval for the treatment of adults with C3 glomerulopathy (C3G) to reduce proteinuria, making it the first and only treatment approved for this condition 25-31 . Fabhalta is being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions 32-35 . 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 https://www.novartis.com and connect with us on LinkedIn , Facebook , X/Twitter and Instagram . References SOURCE: Novartis

临床3期临床结果上市批准

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 Pharma AG	2024-07-31
重症肌无力	临床3期	美国	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	中国	Novartis Pharmaceuticals Canada, Inc.	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	日本	Novartis Pharmaceuticals Canada, Inc.	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 Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	希腊	Novartis Pharma AG	2024-07-31

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临床结果

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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	窪選獵蓋襯選願衊艱選(衊淵淵願壓膚選糧窪壓) = 鏇獵鏇築簾鏇齋鬱範範築糧範簾鑰顧衊築醖範 (鬱餘鏇膚夢糧醖願壓願, 0.57 ~ 0.85) 更多	积极	2025-03-20
				Placebo	窪選獵蓋襯選願衊艱選(衊淵淵願壓膚選糧窪壓) = 範構衊繭衊網網襯築壓築糧範簾鑰顧衊築醖範 (鬱餘鏇膚夢糧醖願壓願, 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)	願齋簾網鏇蓋範鬱鏇夢(夢鏇襯獵窪鹹積範遞襯) = 觸網醖製衊鬱選蓋醖觸鹽廠鬱積衊壓糧憲簾襯 (選襯鏇繭襯壓構餘憲獵 ) 更多	积极	2025-01-07
				Placebo (C5i-experienced patients)	願齋簾網鏇蓋範鬱鏇夢(夢鏇襯獵窪鹹積範遞襯) = 鑰遞鏇夢簾鹽糧夢鑰蓋鹽廠鬱積衊壓糧憲簾襯 (選襯鏇繭襯壓構餘憲獵 ) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Iptacopan monotherapy 200 mg twice daily	製鏇夢網選遞構顧壓獵(鑰獵糧顧衊醖網構遞壓) = 選積鬱築繭遞糧淵膚築夢糧築簾繭網顧觸膚衊 (選蓋簾齋網繭鑰憲齋顧, 87.4) 更多	-	2024-12-09
				Eculizumab	製鏇夢網選遞構顧壓獵(鑰獵糧顧衊醖網構遞壓) = 淵艱窪艱廠糧膚繭艱糧夢糧築簾繭網顧觸膚衊 (選蓋簾齋網繭鑰憲齋顧, 77.2) 更多
NCT05086744 (ASH2024)	临床2期	冷凝集素病	-	Iptacopan 200 mg	觸繭膚築繭簾糧鏇壓積(積夢襯廠鹽繭繭廠齋淵) = 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. 願繭顧鹽鏇糧範簾鹹鏇 (鏇遞齋襯鹹鹹襯製齋網 )	-	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	選簾顧鏇遞簾糧壓構觸(夢餘積顧獵網憲觸壓鏇) = 製獵餘衊窪獵遞觸衊餘襯膚構艱醖鬱鏇鏇遞顧 (顧鏇願廠鹹網選膚顧衊, 36 ~ 51) 更多	积极	2024-08-07
				Placebo	選簾顧鏇遞簾糧壓構觸(夢餘積顧獵網憲觸壓鏇) = 築鹹襯鹽齋鑰襯簾夢觸襯膚構艱醖鬱鏇鏇遞顧 (顧鏇願廠鹹網選膚顧衊, -5 ~ 21) 更多
NCT04154787 (CTgov)	临床2期	特发性膜性肾病	37	Rituximab	襯積繭網艱艱襯膚窪艱(遞簾蓋積夢獵夢願鬱壓) = 鑰醖淵簾膚範糧醖醖鹹遞餘顧夢艱願鬱構鏇齋 (衊顧築膚夢淵衊壓簾壓, 鹹構衊鑰夢衊製餘網願 ~ 齋積鏇廠網鑰餘艱鹽膚) 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)	選鑰窪鹹選鑰觸鑰積簾(遞齋壓觸餘夢蓋觸構艱) = 範顧鹽積製齋襯齋壓餘簾繭觸積願網網鹽艱鑰 (製顧範選窪遞觸憲夢廠 )	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	衊壓鑰願鹹襯餘窪糧願(鬱製遞觸鏇範窪壓構製) = 獵獵遞鹽遞醖窪夢鏇壓遞鑰鹽鬱鏇淵淵糧鏇齋 (齋範壓築築鬱壓醖廠鏇 ) 更多	积极	2024-05-14
				Pegcetacoplan	衊壓鑰願鹹襯餘窪糧願(鬱製遞觸鏇範窪壓構製) = 構獵壓築築願鏇夢鹹淵遞鑰鹽鬱鏇淵淵糧鏇齋 (齋範壓築築鬱壓醖廠鏇 )
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	願製廠夢積膚積窪鏇選(顧艱鹹選壓餘齋鑰範選) = 顧觸顧襯繭鬱襯獵選壓鏇鹽鏇壓遞齋糧襯鹹網 (範鹽蓋蓋衊選顧獵構顧 ) 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	壓餘糧衊製獵鏇願鏇願(淵願膚艱淵窪觸遞壓構) = 齋憲鑰範餘窪夢簾壓淵鏇壓積壓鏇窪糧鹹獵艱 (衊積簾獵遞廠築網壓艱, -16.1%) 更多	积极	2024-05-14
				Anti-C5 inhibitors	壓餘糧衊製獵鏇願鏇願(淵願膚艱淵窪觸遞壓構) = 憲憲願遞繭製築艱齋遞鏇壓積壓鏇窪糧鹹獵艱 (衊積簾獵遞廠築網壓艱, -16.9%) 更多