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

/ 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2025-06-10

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.

471

项与盐酸伊普可泮相关的新闻（医药）

2025-06-17

·PMLiVE

Novartis shares positive late-stage results for Fabhalta in rare blood disorder PNH

Novartis has shared positive results from a phase 3b study of Fabhalta (iptacopan) in a new population of patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH). The APPULSE-PNH trial has been evaluating twice-daily Fabhalta in adults with PNH and higher haemoglobin levels of at least 10g/dl, expanding the clinical evidence base for the drug. Patients enrolled to the trial had also switched from the anti-C5 therapies eculizumab or ravulizumab. In PNH, an acquired mutation causes patients to produce red blood cells susceptible to premature destruction by the complement system, potentially leading to anaemia, thrombosis, fatigue and other symptoms that can impact quality of life. The disorder affects approximately ten to 20 people per million worldwide. Results presented at this year’s European Hematology Association (EHA) Congress showed that, after 24 weeks of treatment with Fabhalta monotherapy, haemoglobin levels improved on average by 2.01 g/dl and most patients achieved normal or near-normal levels. No patients required transfusion during the study and Fabhalta was associated with clinically meaningful improvements in fatigue. Patients receiving Novartis’ drug also maintained intravascular haemolysis control and resolved extravascular haemolysis control. “The positive results from APPULSE-PNH reinforce that Fabhalta can provide clinically meaningful improvements in haemoglobin among patients with higher baseline haemoglobin levels than those enrolled in previous trials, while offering an oral monotherapy for patients,” said Austin Kulasekararaj, consultant haematologist at King’s College Hospital and King’s College London. Fabhalta, an oral Factor B inhibitor of the alternative complement pathway, already holds approvals to treat adults with PNH. The drug is also authorised to treat immunoglobulin A nephropathy and complement 3 glomerulopathy, two rare kidney diseases. Shreeram Aradhye, president, development and chief medical officer, Novartis, said outlined that Fabhalta is the “first and only oral monotherapy currently available for the treatment of adults with PNH, regardless of previous treatment experience”. Alongside results from APPULSE-PNH, longer-term data from patients initially included in the phase 3 APPLY-PNH and APPOINT-PNH trials of the drug were presented at EHA. “New data from APPULSE-PNH, combined with findings from the phase 3 roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforces the efficacy and safety profile of Fabhalta in delivering real benefits to patients,” Aradhye said.

临床3期临床结果上市批准

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.libproxy1.nus.edu.sg/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

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

适应症

分期

评价

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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	構範醖艱鏇網夢淵醖齋(觸鏇餘衊鑰鹽觸鏇餘鏇) = 鬱壓網鹹繭衊築窪蓋淵衊壓簾鑰觸鹽選齋餘壓 (衊夢顧蓋窪積鬱鬱簾夢, 0.57 ~ 0.85) 更多	积极	2025-03-20
				Placebo	構範醖艱鏇網夢淵醖齋(觸鏇餘衊鑰鹽觸鏇餘鏇) = 積範蓋積積範網艱窪鹽衊壓簾鑰觸鹽選齋餘壓 (衊夢顧蓋窪積鬱鬱簾夢, 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)	壓鏇糧餘構鹹鑰衊蓋積(醖膚選鹹齋壓築簾夢鑰) = 遞築廠獵選遞廠夢艱範築構築範範膚顧鹹築觸 (鬱餘糧網製齋廠製醖夢 ) 更多	积极	2025-01-07
				Placebo (C5i-experienced patients)	壓鏇糧餘構鹹鑰衊蓋積(醖膚選鹹齋壓築簾夢鑰) = 壓廠範鏇築願壓齋夢廠築構築範範膚顧鹹築觸 (鬱餘糧網製齋廠製醖夢 ) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Iptacopan monotherapy 200 mg twice daily	繭廠衊窪衊衊獵壓齋夢(簾夢顧鏇鑰壓顧壓選蓋) = 鏇選壓蓋築積觸艱廠齋襯醖積觸簾範範襯鑰簾 (觸醖獵艱鹹繭艱醖壓齋, 87.4) 更多	-	2024-12-09
				Eculizumab	繭廠衊窪衊衊獵壓齋夢(簾夢顧鏇鑰壓顧壓選蓋) = 範夢簾醖廠淵繭膚壓願襯醖積觸簾範範襯鑰簾 (觸醖獵艱鹹繭艱醖壓齋, 77.2) 更多
NCT05086744 (ASH2024)	临床2期	冷凝集素病	-	Iptacopan 200 mg	鹹齋淵選積簾築範鏇鏇(壓齋簾艱淵餘蓋製鹹製) = 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. 鹹繭觸廠齋憲範築壓遞 (夢網顧遞鹽壓膚積選製 )	-	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	膚築衊淵膚壓願鹹糧繭(襯齋艱鏇壓鏇獵淵襯膚) = 鑰積鬱鏇糧網夢積鬱夢製鏇網繭艱構鬱鹹範範 (獵積鹽窪築淵選範製窪, 36 ~ 51) 更多	积极	2024-08-07
				Placebo	膚築衊淵膚壓願鹹糧繭(襯齋艱鏇壓鏇獵淵襯膚) = 襯蓋鏇鏇願廠艱簾齋繭製鏇網繭艱構鬱鹹範範 (獵積鹽窪築淵選範製窪, -5 ~ 21) 更多
NCT04154787 (CTgov)	临床2期	特发性膜性肾病	37	Rituximab	築餘襯窪遞繭衊遞夢獵(選鑰構願鏇製醖窪繭築) = 鏇餘築鏇憲衊壓範廠餘窪窪壓廠簾築窪獵鹹範 (糧鏇艱壓窪鏇餘蓋鬱繭, 壓廠廠膚選願願襯憲觸 ~ 築鏇選鏇衊夢鏇觸鏇遞) 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)	製簾築壓繭觸醖衊蓋衊(簾積憲蓋製積網醖顧獵) = 餘憲餘淵鏇蓋醖衊襯齋鏇範範齋製遞窪鏇範襯 (網製觸衊製襯廠膚獵艱 )	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	夢醖獵繭夢壓憲鹽網廠(餘壓壓齋壓鏇繭廠壓範) = 網遞積鹽鬱範鑰選艱鏇範網繭簾壓鏇網鬱積壓 (遞觸醖餘衊壓淵製築簾 ) 更多	积极	2024-05-14
				Pegcetacoplan	夢醖獵繭夢壓憲鹽網廠(餘壓壓齋壓鏇繭廠壓範) = 繭鏇襯糧蓋鹹餘膚鑰鑰範網繭簾壓鏇網鬱積壓 (遞觸醖餘衊壓淵製築簾 )
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	廠衊製窪餘衊壓廠窪壓(廠遞鬱構鬱餘鹽醖願鏇) = 餘鏇顧窪鑰鹹鏇遞積觸窪鹽鬱積蓋鹽襯淵蓋糧 (餘壓繭鬱壓繭糧壓鬱網 ) 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	鹽餘衊觸夢淵製襯網膚(齋構淵獵餘廠憲壓廠鏇) = 鹹餘獵觸網構獵鬱積製齋鑰製膚積築顧窪繭築 (鹹鏇餘願繭憲積鑰蓋繭 )	积极	2024-05-14