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.

CTIS2023-504550-35-00

/ Recruiting临床3期

A multicenter, single arm, open-label study to evaluate efficacy and safety of switching from anti-C5 antibody therapy to iptacopan therapy in study participants with aHUS - CLNP023F12302

开始日期2025-04-03

申办/合作机构

Novartis Pharma AG

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）

2025-05-01·PHARMACOECONOMICS

Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria

Review

作者: Reeve, Alex ; Vydyula, Krishna ; Youngs, Matthew ; Gallacher, Daniel ; Donoghue, Jeremiah ; Kunst, Natalia ; Trikha, Roochi

In 2024, the National Institute for Health and Care Excellence (NICE) recommended two new health technologies for paroxysmal nocturnal haemoglobinuria. This review systematically compares the clinical and cost-effectiveness evidence considered within the NICE single technology appraisals of iptacopan, danicopan and pegcetacoplan, examines the consistency of the clinical evidence and economic modelling, and considers whether single technology appraisals are a suitable apparatus for consistent decision making. The studies used different follow-up lengths and used different definitions for reporting breakthrough haemolysis (BTH), but otherwise reported similar outcomes and found a significant benefit for their interventions. A lack of direct evidence and unreliable indirect comparisons meant that naïve comparisons across trials were carried into the economic modelling despite differences in their control arms. Approaches to modelling BTH and associated dose escalation differed across appraisals, despite information for pegcetacoplan coming from the same source in each appraisal, which had a large impact on the economic results. This review raises the question of whether NICE should implement multiple technology appraisals more frequently to reduce these inconsistences. Additionally, we recommend the development of a framework for revisiting positive recommendations when the implementation of health technologies deviates from assumptions made in the economic modelling to ensure cost-effective healthcare is preserved.

2025-05-01·CLINICAL PHARMACOLOGY & THERAPEUTICS

Effect of Target‐Mediated Disposition on Iptacopan Clinical Pharmacokinetics in Participants with Normal or Impaired Hepatic Function

Article

作者: Lawitz, Eric ; Baltcheva, Irina ; Weis, Wendy ; Shah, Bharti ; Fink, Martin ; Schmouder, Robert ; Kulmatycki, Kenneth ; Burmeister Getz, Elise ; Stein, Richard R.

Iptacopan, a first‐in‐class complement factor B inhibitor acting proximally in the alternative complement pathway, has been shown to be safe and effective for patients with complement‐mediated diseases. Iptacopan selectively binds with high affinity to factor B, a soluble, plasma‐based, hepatically produced protein. Factor B is abundant in the circulation but can be saturated at the iptacopan clinical dose of 200 mg twice daily. Iptacopan pharmacokinetics (PK) are influenced by target binding. This target‐mediated drug disposition (TMDD) behavior makes PK data useful for understanding target occupancy and motivates modeling of drug‐target binding to connect exposure with pharmacological effect. A phase I hepatic impairment (HI) PK study measuring both total and unbound iptacopan PK profiles provided an opportunity to characterize the effect of variation in target concentration (due to varying hepatic function) on iptacopan PK. HI caused no change in total iptacopan exposure but increased unbound iptacopan exposure 1.38‐ to 3.72‐fold in participants with mild, moderate, or severe HI relative to demographically matched participants with normal hepatic function, with the largest increases in severe HI. A two‐site competitive binding model was developed to elucidate the relationship between iptacopan PK and factor B occupancy to characterize exposure thresholds for maximal target engagement. The model was used to assess alternative dose regimens to provide insight into how to approach dose recommendations for patients with severe HI. This study provides an example of small‐molecule TMDD, a behavior typically associated with targeted biologics; its importance is too often underappreciated in small‐molecule drug development.

2025-04-22·Blood Advances

Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies

Article

作者: de Castro, Carlos ; Scheinberg, Phillip ; Peffault de Latour, Régis ; Maciejewski, Jaroslaw P. ; Bermann, Georgina ; Kulasekararaj, Austin ; Kumar, Rakesh ; Han, Bing ; Dahlke, Marion ; Vallow, Susan ; Risitano, Antonio M. ; Ueda, Yasutaka

Abstract:

Iptacopan, a first-in-class, oral, selective complement factor B inhibitor, demonstrated efficacy and safety as monotherapy in C5 inhibitor (C5i)–experienced (APPLY-PNH; NCT04558918) and C5i-naive (APPOINT-PNH; NCT04820530) patients with paroxysmal nocturnal hemoglobinuria (PNH). In the APPLY-PNH and APPOINT-PNH trials, changes in fatigue (Functional Assessment of Chronic Illness Therapy–Fatigue [FACIT-Fatigue]) and health-related quality of life (HRQOL; European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire [EORTC QLQ-C30]) from baseline to day 168 were evaluated. The proportion of patients achieving meaningful within-patient change (MWPC) on the FACIT-Fatigue and 4 EORTC QLQ-C30 subscales was evaluated using anchor-based thresholds, and correlations between FACIT-Fatigue scores, lactate dehydrogenase (LDH), and hemoglobin (Hb) levels were assessed. In APPLY-PNH (iptacopan, n = 62; C5i, n = 33), more patients in the iptacopan versus the C5i group reached the MWPC threshold for FACIT-Fatigue (51% vs 11%). More patients achieved MWPC on EORTC QLQ-C30 subscales in the iptacopan group (39%-49%) versus the C5i group (9%-20%). In APPOINT-PNH (N = 40), 56% achieved MWPC on the FACIT-Fatigue, and the proportion of patients who achieved MWPC on the EORTC QLQ-C30 ranged from 41% to 55%. In C5i-experienced patients, increased Hb levels correlated with improvement in FACIT-Fatigue scores (R = 0.48); in C5i-naive patients, increased Hb (R = 0.42) and decreased LDH (R = −0.53) (all P < .001) correlated with improved FACIT-Fatigue scores. C5i-experienced and -naive patients receiving iptacopan exhibited meaningful improvement in fatigue, HRQOL, and disease-related symptoms, which correlated with clinical improvement in hematologic markers of disease control.

448

项与盐酸伊普可泮相关的新闻（医药）

2025-05-15

·GlobeNewswire-Health Care

New Novartis data at ASCO and EHA showcase momentum of pioneering portfolio with promising pipeline

NATALEE subanalysis evaluates Kisqali in pre-menopausal early breast cancer patients, amid rising diagnosis rates in younger patients Pluvicto analysis and Scemblix ASC4START primary endpoint results provide insights into use in earlier settingsFabhalta APPULSE-PNH full results build on Phase III program, reporting new data from expanded PNH population in adults switching from anti-C5Ianalumab Phase II data in immune thrombocytopenia and longer-term pelabresib Phase III data in myelofibrosis show breadth of pipeline in hematologic diseases Basel, May 15, 2025 – Novartis will present data from 60 company or investigator sponsored abstracts that have the potential to change clinical practice, at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and the European Hematology Association (EHA) 2025 Congress. “The breadth of our oncology and hematology portfolio – anchored by Kisqali, Pluvicto, Scemblix and Fabhalta – demonstrates our leadership in both solid tumors and hematologic diseases,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “At ASCO and EHA, we will present new data on these priority medicines as well as updates from our pipeline and our industry-leading radioligand therapy research.” Novartis will also highlight its US partnerships with the National Football League (NFL), Alliance for Breast Cancer Policy, and ZERO Prostate Cancer, which encourage people to make proactive decisions about their health and advance patient-centered policy solutions to help improve outcomes. “We're witnessing a profound shift in how people move through their cancer journey, with cancer diagnoses occurring at younger ages and, simultaneously, older patients living longer and approaching aging with new vigor,” said Victor Bultó, President, US, Novartis. “As a leader in driving medical advances in oncology, we have the responsibility to also make a difference in areas beyond treatment innovation. By partnering across the ecosystem, our goal is to advance the conversation around earlier detection and meet the evolving needs of this next generation of cancer patients.” Key highlights of data accepted by ASCO include: Medicine Abstract Title Abstract Number/ Presentation Details Kisqali®(ribociclib)* Efficacy and safety of ribociclib (RIB) + nonsteroidal aromatase inhibitor (NSAI) in NATALEE: Analysis across menopausal status and age Abstract #516Rapid OralJune 1, 8:00 – 9:30am CDT Kisqali®(ribociclib) Real-world (RW) analysis of characteristics and risk of recurrence (ROR) in Black patients (pts) with HR+/HER2− early breast cancer (EBC) eligible for NATALEE Abstract #527Poster PresentationJune 2, 9:00am – 12:00pm CDT Kisqali®(ribociclib) Adjuvant WIDER: A phase 3b trial of ribociclib (RIB) + endocrine therapy (ET) as adjuvant treatment (tx) in a close-to-clinical-practice patient (pt) population with HR+/HER2− early breast cancer (EBC) Abstract #TPS617Poster PresentationJune 2, 9:00am – 12:00pm CDT Kisqali®(ribociclib) First-line (1L) ribociclib (RIB) + endocrine therapy (ET) vs combination chemotherapy (combo CT) in clinically aggressive hormone receptor (HR)+/HER2− advanced breast cancer (ABC): A subgroup analysis of patients (pts) with or without liver metastases (mets) from RIGHT Choice Abstract #1069Poster PresentationJune 2, 9:00am – 12:00pm CDT Scemblix®(asciminib) Efficacy and safety of asciminib (ASC) in patients (pts) with chronic-phase chronic myeloid leukemia (CML-CP) after 1 tyrosine kinase inhibitor (TKI): Interim analysis (IA) of the phase 2 ASC2ESCALATE trial Abstract #6516Rapid OralMay 30, 1:00 – 2:30pm CDT Scemblix® (asciminib) Primary endpoint results of the phase 3b ASC4START trial of asciminib (ASC) vs nilotinib (NIL) in newly diagnosed chronic phase chronic myeloid leukemia (CML-CP): Time to treatment discontinuation due to adverse events (TTDAE) Abstract #6501Oral PresentationJune 2, 3:00 – 6:00pm CDT Pluvicto®(lutetium Lu 177 vipivotide tetraxetan) Clinical outcomes of prompt versus deferred 177Lu-PSMA-617 initiation for metastatic castration-resistant prostate cancer (mCRPC) based on prior androgen receptor pathway inhibitor (ARPI) and taxane chemotherapy exposure: a real-world PRostatE Cancer dISease observatION (PRECISION) data platform analysis Abstract #e17030Online Publication Pluvicto®(lutetium Lu 177 vipivotide tetraxetan) Real-world outcomes among patients with metastatic castration-resistant prostate cancer (mCRPC) receiving guideline-recommended therapies after treatment with 177Lu-PSMA-617: a real-world PRostatE Cancer dISease observatION (PRECISION) data platform analysis Abstract #e17035Online Publication Pluvicto®(lutetium Lu 177 vipivotide tetraxetan) PSMA-delay castration (DC): An open-label, multicenter, randomized phase 3 study of [177Lu]Lu-PSMA-617 versus observation in patients with metachronous PSMA-positive oligometastatic prostate cancer (OMPC) Abstract #TPS5127Poster PresentationJune 2, 9:00am – 12:00pm CDT Key highlights of data accepted by EHA include: Medicine Abstract Title Abstract Number/ Presentation Details Fabhalta®(iptacopan) APPULSE-PNH: Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin (Hb) increases in patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and Hb ≥10 g/dL on anti-C5 therapy Abstract #S183Oral PresentationJune 13, 5:00 – 6:15pm CEST Fabhalta®(iptacopan) The 2-year safety and efficacy of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH) from APPLY- and APPOINT-PNH studies who entered the roll-over extension program (REP) Abstract #PF660Poster PresentationJune 13, 6:30 – 7:30pm CEST Scemblix®(asciminib) Asciminib (ASC) shows superior tolerability vs nilotinib (NIL) in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): Primary endpoint results of the phase (Ph) 3b ASC4START trial Abstract #S166Oral PresentationJune 13, 5:00 – 6:25pm CEST Scemblix®(asciminib) Improved patient-reported outcomes (PROs) with asciminib (ASC) vs investigator-selected tyrosine kinase inhibitors (IS-TKIs) in newly diagnosed chronic myeloid leukemia (CML): ASC4FIRST wk 48 analysis Abstract #PS1588Poster PresentationJune 14, 6:30 – 7:30pm CEST Scemblix®(asciminib) Interim analysis (IA) results from ASC2ESCALATE support asciminib (ASC) as a treatment (Tx) option in chronic-phase chronic myeloid leukemia (CML-CP) after 1 tyrosine kinase inhibitor (TKI) Abstract #PF595Poster PresentationJune 13, 6:30 – 7:30pm CEST Pelabresib(DAK539) Pelabresib in combination with ruxolitinib for janus kinase inhibitor-naive patients with myelofibrosis: 72-week follow-up with long-term efficacy outcomes of the phase III MANIFEST-2 study Abstract #S223Oral PresentationJune 12, 5:00 – 6:15pm CEST Ianalumab(VAY736) A Phase 2 Study of Ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapy (VAYHIT3) Abstract #S312Oral PresentationJune 15, 11:00am – 12:15pm CEST Rapcabtagene autoleucel (YTB323) Rapcabtagene Autoleucel (YTB323) in patients with relapsed/refractory diffuse large B-cell lymphoma: A phase II trial clinical update Abstract #PF1152Poster PresentationJune 13, 6:30 – 7:30pm CEST Novartis in oncologyThe Novartis oncology strategy focuses on people living with cancer and those who care for them, from loved ones to clinical care teams, including their providers. For the past 30+ years, the aim has been to extend and improve lives by discovering differentiated, innovative and practice-changing medicines for patients. As Novartis reimagines medicine, it collaborates with a wide range of patient advocacy groups and supports education, early cancer screening and diagnosis, all while innovating at a rate that is unparalleled in the industry. With approximately 35 research and development projects across solid tumors, hematology and radioligand therapy (RLT), Novartis is committed to using technology, leading science and patient-centered research to deliver pioneering cancer care for all those in need. DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. # # # *Kisqali was developed by Novartis under a research collaboration with Astex Pharmaceuticals. Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

临床2期ASCO会议临床3期临床结果

2025-05-15

·CPHI制药在线

金笔奖|从罕见病到常见病，补体药物开疆拓土

关注并星标CPHI制药在线在创新药市场中，补体药物是一个重要领域，也是研发壁垒较高的一个赛道。但鉴于其拥有巨大的应用前景，近年来补体药物的研发进入快车道，其适应症范围也在逐步拓展。近日，诺华的补体因子B（CFB）抑制剂伊普可泮（Iptacopan，Fabhalta）获NMPA批准，用于成人C3肾小球病（C3G）的治疗，这是补体药物在肾病治疗领域解锁的又一块拼图。该适应症的获批，彰显了补体药物的巨大潜力，同时也将其研发热度再次掀起。01伊普可泮的适应症版图伊普可泮是一款同类首创的CFB抑制剂，通过与补体旁路途径中的FB结合，调节C3的裂解、下游效应物的产生和末端途径的放大，控制C3b介导的血管外溶血和末端补体介导的血管内溶血。2023年12月，伊普可泮首次获FDA批准，治疗成人阵发性睡眠性血红蛋白尿症（PNH）。PNH是一种罕见的血液系统疾病，发病率约为每百万人口1～10例，已被纳入我国《第一批罕见病目录》。该病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，虽为良性血管内溶血性疾病，但严重者可危及生命。在补体抑制剂获批前，PNH主要以支持治疗为主，包括红细胞输注、补充造血原料、糖皮质激素和雄激素的使用以及免疫抑制剂的应用。但是传统支持治疗疗效有限，多数患者无法摆脱输血依赖，且患者10年总生存率仅70.7%~77.6%，生存率及生活质量亟待提高。伊普可泮的上市，改变了PNH的治疗局面。在APPOINT-PNH研究中，在未接受过补体抑制剂治疗的成人PNH患者中，给予伊普可泮单药治疗后，92%的患者在未输注红细胞的情况下血红蛋白水平相较基线增加≥2g/dL，近2/3患者的血红蛋白恢复至正常水平（≥12g/dL），98%的患者避免输血。同时，患者的疲劳症状表现出具有临床意义的改善。而且伊普可泮避免患者不断往返于居住地和医院，提高了患者依从性，使PNH从一个几乎不可治愈、危及生命的严重疾病转变为可接受长期管理的慢性疾病。2024年8月，FDA再次批准伊普可泮新适应症，用于降低成人IgA肾病（IgAN）患者的蛋白尿水平，这些患者有疾病迅速进展的风险。IgAN是一种罕见的异质性、进行性肾脏疾病。全世界每年每百万人中约有25人被新诊断为IgAN，由于患者具有持续较高水平的蛋白尿，因此可能在10年内进展为肾衰竭。在III期APPLAUSE-IgAN研究中，与安慰剂组相比，伊普可泮组患者的蛋白尿水平减少了38.3%（p<0.0001），并减缓了肾功能下降速度。而且伊普可泮的安全性和耐受性良好，与既往研究一致。伊普可泮的这一突破性进展，为IgA肾病的精准治疗开辟了新途径。2025年3月，伊普可泮获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。C3G是一种极其罕见的进行性肾脏疾病，多见于儿童和年轻人。每年全球每百万人中约有1-2人确诊。大约一半的C3G患者在诊断后10年内进展为肾衰竭，需要终身透析和/或肾移植。在III期APPEAR-C3研究中，评估了伊普可泮（200mg，每日2次）在原发性C3G患者中的疗效和安全性。结果显示，伊普可泮可迅速降低蛋白尿，最早在第14天见效，并持续至第12个月。同时，伊普可泮在研究中也表现出了良好的耐受性和安全性。伊普可泮自上市以来，短短两年多时间，已顺利获批三项适应症，曾被行业媒体Evaluate列为10款值得关注的潜在重磅疗法之一。02打破治疗困局，补体药物围攻罕见病补体系统是人体先天免疫系统的一部分，由大约50种蛋白质及蛋白质片段组成，主要由肝脏细胞和巨噬细胞产生，具有增强抗体、吞噬细胞、清除生物体中的微生物和受损细胞、攻击入侵病原体等能力。正常情况下，补体处于无活性状态，只有被某些因子激活后才产生具有生物活性的裂解片段，作用于相应靶细胞的受体而发挥生物学效应。补体被活化后产生大量具有活性的裂解产物如：C1q、C3a、C3b、C5a、C5b-9等。这些裂解产物有介导细胞溶解、调节细胞吞噬、清除免疫复合物和损伤凋亡细胞等生物学效应。补体激活途径主要有三条：经典激活（Classical pathway），凝集素激活（Lectin pathway）和旁路激活（Alternative pathway）。经典激活是由抗原抗体复合物结合C1q启动；凝集素激活发生在感染早期，机体炎症反应诱导肝细胞释放急性期蛋白MBL，C反应蛋白等。旁路激活是指病原微生物直接从C3激活。由于补体网络极其复杂，他在激活与调控之间维持着精妙的平衡，因此补体的选择性抑制十分重要，相关药物研发因此困难重重。目前已获批上市的补体药物有10款，适应症多集中于PNH等罕见病。2007年，由Alexion（于2020年被阿斯利康收购）研发的Soliris（eculizumab）获批上市，是全球获批的首款C5补体抑制剂，也成为了当时PNH首个也是唯一的治疗药物。Soliris成为了全球最畅销的罕见病药物之一。目前，除PNH外，Soliris已批准多种罕见病，包括：非典型溶血尿毒症综合征（aHUS）、全身型重症肌无力（gMG）、视神经脊髓炎谱系障碍（NMOSD）。2018年，阿斯利康的第二代、长效C5补体抑制剂Ultomiris（ravulizumab）获FDA批准上市。与Soliris相比，Ultomiris每年需要的输液次数减少，并具有良好的疗效和安全性。目前该药已批准的适应症包括PNH和aHUS。Ultomiris的业绩依然不容小觑，2020年销售额就达到了10.77亿美元。在阿斯利康两款重磅药物影响下，投入补体药物研发的药企越来越多。从2021年开始，补体药物研发进入快车道。2021年5月，Apellis Pharmaceuticals的C3补体抑制剂Empaveli（pegcetacoplan）获FDA批准上市，用于治疗PNH成人患者，成为首个FDA批准的用于治疗PNH的C3靶向疗法。2021年9月，安进和CSL Vifor共同开发的阿伐可泮（avacopan）获FDA批准上市，用于联合包括糖皮质激素在内的标准治疗方案治疗成人活动性严重ANCA相关血管炎（MPA和GPA）。阿伐可泮是一种补体5a受体（C5aR）拮抗剂，可抑制C5aR和过敏毒素C5a之间的相互作用，阻断C5a介导的中性粒细胞激活和迁移。阿伐可泮是FDA批准的首款口服补体5a受体拮抗剂。2022年，赛诺菲的补体C1s单抗Enjaymo(Sutimlimab)获FDA批准上市，用于冷凝集素病（CAD)患者降低由于溶血导致的红细胞输注需求。Sutimlimab通过阻断经典补体途径中C1s蛋白的功能而发挥抑制溶血的作用，是FDA批准的首款用于治疗CAD的补体C1s抗体。此后几年，安斯泰来的Izervay（avacincaptad pegol）、再生元的Veopoz（Pozelimab）、UCB的Zilbrysq相继获批上市，其中Izervay是一款旨在抑制补体C5蛋白的聚乙二醇化RNA适配体；Veopoz则是一款新一代补体C5单抗；Zilbrysq是一款新型大环肽类C5补体抑制剂。总之，目前已上市的补体药物大多采用从罕见血液疾病切入的研发策略，逐步向常见病领域跃迁。03从罕见病到常见病，补体药物厚积薄发由于补体系统在免疫调节、炎症反应等过程中都起着重要作用，近年来，补体药物不断向常见病领域探索。2023年，Apellis Pharmaceuticals的pegcetacoplan获FDA批准，成为第一款治疗地图样萎缩（GA）的药物。GA是干性年龄相关性黄斑变性（AMD）的一种晚期形式。AMD是一种影响眼后部部分黄斑的疾病，一些AMD患者最终会发展为GA。GA可导致进行性和永久性视力丧失。而且如果患者一只眼睛患有GA，则很有可能在另一只眼睛中也患上该疾病，严重影响患者生活质量。据悉，美国近2000万成年人患有某种形式的年龄相关性AMD，其中有100万人患有GA。全球GA患者人数估计超过500万。Pegcetacoplan在GA上的突破，标志着补体药物在常见病领域的破冰。2025年2月，安斯泰来宣布FDA批准其Izervay用于治疗GA的补充新药申请（sNDA）。经此次批准，Izervay的给药持续时间再无限制——为医生和患者管理GA提供了更大的灵活性。这些成果验证了补体系统在视网膜疾病治疗中的作用，全方位展示补体药物的广泛治疗益处。在经过数十年的探索后，补体药物的开发终于迎来了快速爆发期，靶点从C5拓展到C3、C5a、C1s、CFB等，药物类型也从单克隆抗体拓展到环肽、小分子、核酸药物等，适应症更是从罕见病向常见病拓展。总的来说，补体药物的研发虽然困难，但非常值得持续探索。主要参考资料： 1.https://www.novartis.com/sites/novartis_com/files/q2-2024-investor-presentation.pdf.2.Risitano AM, Röth A, Soret J, et al. Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. Lancet Haematol. 2021;8(5):e344-e354.3.Peffault de Latour R, Röth A, Kulasekararaj AG, et al. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2024 Mar 14;390(11):994-1008. 作者简介:@小饼，药学硕士，现为疾控工作人员，每天游走于纷繁复杂的数据里，深感自己渺小的同时，又庆幸能够见证中国生物医药发展的黄金时代。希望和各位共同学习、提高。END【企业推荐】领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

临床结果临床3期申请上市

2025-05-08

·PRNewswire

LUPKYNIS Strengthens Market Position as Demand for Lupus Nephritis Treatment Grows | DelveInsight

As a novel calcineurin inhibitor with proven efficacy in improving renal outcomes, LUPKYNIS addresses a high unmet medical need. With increasing awareness, supportive guidelines, and potential global expansion, it is well-positioned for steady market growth. LAS VEGAS, May 8, 2025 /PRNewswire/ -- DelveInsight's " LUPKYNIS Market Size, Forecast, and Market Insight Report" highlights the details around LUPKYNIS, the first oral lupus nephritis-specific treatment, which has expanded the therapeutic arsenal. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of LUPKYNIS. The report also highlights the historical and forecasted sales from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Aurinia Pharmaceuticals/Otsuka Pharmaceutical's LUPKYNIS (voclosporin) Overview LUPKYNIS (voclosporin) is an oral immunosuppressant classified as a calcineurin inhibitor (CNI), shown to improve both short- and long-term outcomes in adult patients with active lupus nephritis when used alongside standard immunosuppressive therapies. By targeting calcineurin, LUPKYNIS helps suppress cytokine activity, inhibits interleukin-2 (IL-2) production, and reduces T-cell driven immune responses. Although the exact mechanism by which voclosporin inhibits calcineurin is not fully understood, it is known that lymphocyte activation increases intracellular calcium, which binds to calcineurin's regulatory site. This activates the calmodulin-binding catalytic subunit, leading to dephosphorylation and activation of the transcription factor NFATc (nuclear factor of activated T-cells, cytoplasmic). The drug's immunosuppressive effect results in decreased lymphocyte proliferation, reduced cytokine production, and lower expression of T-cell activation markers. The recommended initial dose of LUPKYNIS is 23.7 mg twice daily. It should be used in conjunction with mycophenolate mofetil (MMF) and corticosteroids. Learn more about LUPKYNIS projected market size for lupus nephritis @ LUPKYNIS Market Potential Lupus nephritis is a serious and potentially life-threatening complication of systemic lupus erythematosus (SLE), affecting 40–60% of those diagnosed with the disease. It is typically characterized by symptoms such as hematuria (blood in the urine) and proteinuria (protein in the urine). This condition is more prevalent in women, particularly between the ages of 20 and 40. In 2023, the United States recorded the highest number of lupus nephritis cases among the 7MM, with approximately 211K cases, a figure projected to grow by 2034. Treatment strategies for lupus nephritis depend on the kidney biopsy classification of the disease. Standard therapy often includes corticosteroids in combination with immunosuppressive drugs like cyclophosphamide, mycophenolate mofetil, azathioprine, and calcineurin inhibitors. Rituximab, a monoclonal antibody, is widely used, especially for patients with relapsing or treatment-resistant LN, due to its ability to reduce reliance on steroids. Currently, there are only two FDA-approved medications for lupus nephritis: BENLYSTA (belimumab), available as an intravenous or subcutaneous formulation, and LUPKYNIS (voclosporin), a novel oral calcineurin inhibitor. Leading pharmaceutical companies such as Novartis, AstraZeneca, Roche, and Kezar Life Sciences, among others, are actively developing innovative therapies that could redefine the treatment paradigm for lupus nephritis. The treatment landscape is rapidly evolving, shifting away from broad immunosuppression toward more precise, targeted approaches. Recent drug approvals and a strong pipeline of candidates highlight both advancements and the ongoing challenges in creating treatments that are effective, safe, and long-lasting. Emerging therapies ranging from anti-CD20 antibodies and complement inhibitors to interferon-blocking agents and CAR-T cell therapies hold promise but must address key issues such as safety, sustained efficacy, and accessibility. As our understanding of the disease's underlying mechanisms continues to grow, the future of lupus nephritis treatment is expected to become increasingly personalized, offering renewed hope to patients battling this complex disorder. Discover more about the lupus nephritis market in detail @ Lupus Nephritis Market Report Emerging Competitors of LUPKYNIS The developing pipeline for lupus nephritis treatments shows a growing variety of approaches. Multiple candidates, both in early and late stages of development, are underway, including ianalumab (VAY736) (Novartis), GAZYVA (obinutuzumab) (Roche), SAPHNELO (anifrolumab) (AstraZeneca), FABHALTA (iptacopan) (Novartis), ULTOMIRIS (ravulizumab) (AstraZeneca), as well as advanced cellular therapies like CABA-201 (Cabaletta Bio) and YTB323 (Novartis). In March 2025, the FDA accepted a supplemental Biologics License Application based on data from the Phase III REGENCY trial, where obinutuzumab demonstrated a 46.4% Complete Renal Response (CRR), compared to 33.1% for placebo. Unlike previous anti-CD20 therapies such as rituximab, it provides enhanced B-cell depletion and is the first in its class to show efficacy in a randomized Phase III lupus nephritis trial. If approved, it will become the first CD20-targeted treatment specifically indicated for lupus nephritis, with anticipated sales of USD 400 million by 2034. To know more about the number of competing drugs in development, visit @ LUPKYNIS Market Positioning Compared to Other Drugs Key Milestones of LUPKYNIS In September 2024, Japan's Ministry of Health approved it for Lupus Nephritis treatment alongside mycophenolate mofetil. In September 2022, the European Commission approved LUPKYNIS for active Class III, IV, or V Lupus Nephritis. In January 2021, Aurinia Pharmaceuticals received US FDA approval for LUPKYNIS to treat adult lupus nephritis patients in combination with immunosuppressive therapy. Discover how LUPKYNIS is shaping the lupus nephritis treatment landscape @ LUPKYNIS Lupus LUPKYNIS Market Dynamics LUPKYNIS, developed by Aurinia Pharmaceuticals, is the first FDA-approved oral therapy specifically indicated for active lupus nephritis, a serious manifestation of systemic lupus erythematosus. Approved in January 2021 in the US, LUPKYNIS entered a previously underserved market where treatment was largely dependent on off-label use of immunosuppressants like mycophenolate mofetil and corticosteroids. The drug represents a significant advancement in lupus nephritis care, offering both improved efficacy and a better safety profile compared to existing options, especially in reducing proteinuria and preserving kidney function. The market dynamics for LUPKYNIS are shaped by several key factors. On the positive side, there is a strong unmet medical need, a growing prevalence of lupus nephritis, and increasing physician awareness about early diagnosis and intervention. Moreover, LUPKYNIS benefits from a relatively high pricing strategy, supported by its novel mechanism and disease-modifying potential. Aurinia has also built a dedicated commercial infrastructure and patient support programs to drive uptake and adherence. However, LUPKYNIS faces competitive and reimbursement challenges. One of the main competitors is BENLYSTA (belimumab) from GSK, which gained FDA approval for lupus nephritis in December 2020, just a month before LUPKYNIS. Benlysta has the advantage of brand recognition and a longer track record in treating systemic lupus. Additionally, payers have shown some hesitancy in approving LUPKYNIS due to its cost, requiring extensive prior authorizations and documentation of clinical need. Patient compliance also remains an issue, given the need for frequent monitoring of kidney function and drug levels. Looking ahead, the market potential for LUPKYNIS will depend on its ability to expand its label, demonstrate long-term real-world effectiveness, and potentially enter international markets. Aurinia is also exploring lifecycle management strategies, including combination therapies and expanded indications. Partnerships or acquisitions could further boost market access and penetration. Overall, while LUPKYNIS is a pioneering therapy with strong clinical value, its commercial success will be defined by navigating payer landscapes, physician adoption, and sustained differentiation from competing therapies. Dive deeper to get more insight into LUPKYNIS's strengths & weaknesses relative to competitors @ LUPKYNIS Market Drug Report Table of Contents Related Reports Lupus Nephritis Market Lupus Nephritis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key lupus nephritis companies, including Hoffmann-La Roche, Chugai Pharmaceutical, Novartis Pharmaceuticals, among others. Lupus Nephritis Pipeline Lupus Nephritis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key lupus nephritis companies, including Hoffmann-La Roche, Qilu Pharmaceutical, Vera Therapeutics, Kyverna Therapeutics, Cabaletta Bio, Takeda, Nkarta Therapeutics, Annexon, Century Therapeutics, Lepton Pharmaceuticals, Transcenta Holding, Inflection Biosciences, among others. Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SLE companies, including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Pipeline Systemic Lupus Erythematosus Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Yake Biotechnology, UCB, Sorrento Therapeutics, SinoMab Bioscience Ltd, Shanghai Junshi Biosciences, Sareum, Sanofi, Roche, Rheos Medicine, Resolve, Provention Bio, Pfizer, Novartis, Neovacs, Merck, Medsenic, Landos Biopharma, Kezar Life Sciences, Kangpu Biopharmaceuticals, Janssen Research & Development, Janssen, InnoCare, ImmuPharma, I-MAB Biopharma, ILTOO, Idorsia Pharmaceuticals, Horizon Therapeutics, Genovax, Exinda Thearapeutics, Equillium, Eli Lilly and Company, Eisai, Daiichi Sankyo Company, Corestem, Corbus Pharmaceuticals, Citryll BV, Chipscreen Biosciences, Carna Bioscience, Bristol-Myers Squibb, Brickell Biotech, Boston Pharmaceuticals, Biogen, Athos Therapeutics, Asahi Kasei Pharma, Aria Pharmaceuticals, Antengene Therapeutics, Amgen, Alpine Immune Sciences, Akeso Biopharma, AbbVie, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准临床3期临床结果免疫疗法

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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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	鏇糧衊選淵鑰齋窪醖糧(願壓壓繭壓壓夢廠鏇簾) = 齋構繭窪獵衊願選憲鹹簾壓鏇鏇鬱憲醖艱選鑰 (糧獵鬱繭構網廠淵齋艱, 0.57 ~ 0.85) 更多	积极	2025-03-20
				Placebo	鏇糧衊選淵鑰齋窪醖糧(願壓壓繭壓壓夢廠鏇簾) = 觸繭選夢廠夢願壓鏇遞簾壓鏇鏇鬱憲醖艱選鑰 (糧獵鬱繭構網廠淵齋艱, 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)	觸顧顧憲獵壓遞網襯醖(憲窪顧製衊廠蓋廠網壓) = 糧膚憲願鬱醖鏇繭製艱願遞餘鑰憲鏇簾鏇齋繭 (襯餘衊簾艱顧鹹壓淵壓 ) 更多	积极	2025-01-07
				Placebo (C5i-experienced patients)	觸顧顧憲獵壓遞網襯醖(憲窪顧製衊廠蓋廠網壓) = 鑰餘積築鹹構範願衊醖願遞餘鑰憲鏇簾鏇齋繭 (襯餘衊簾艱顧鹹壓淵壓 ) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Iptacopan monotherapy 200 mg twice daily	築壓淵積觸窪願醖鏇製(糧壓選簾鑰蓋鹹積選餘) = 蓋鹽構願鹹鑰憲廠淵齋艱膚範夢獵壓簾膚網艱 (遞醖衊醖壓築遞選蓋窪, 87.4) 更多	-	2024-12-09
				Eculizumab	築壓淵積觸窪願醖鏇製(糧壓選簾鑰蓋鹹積選餘) = 衊夢觸鏇壓蓋顧壓鬱遞艱膚範夢獵壓簾膚網艱 (遞醖衊醖壓築遞選蓋窪, 77.2) 更多
NCT05086744 (ASH2024)	临床2期	冷凝集素病	-	Iptacopan 200 mg	積廠廠繭網醖蓋積遞餘(鑰衊窪觸鬱壓顧廠顧鏇) = 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. 觸選願蓋衊繭獵築艱簾 (廠艱壓鑰鏇構艱蓋夢蓋 )	-	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	憲艱糧製鹹醖窪獵網獵(構積鏇選願窪窪鹽襯窪) = 蓋範齋繭淵鑰糧壓窪鏇製淵窪廠選顧鏇選廠衊 (積鹹繭壓鹽鹽遞鏇憲願, 36 ~ 51) 更多	积极	2024-08-07
				Placebo	憲艱糧製鹹醖窪獵網獵(構積鏇選願窪窪鹽襯窪) = 壓繭憲鹹積糧夢夢遞願製淵窪廠選顧鏇選廠衊 (積鹹繭壓鹽鹽遞鏇憲願, -5 ~ 21) 更多
NCT04154787 (CTgov)	临床2期	特发性膜性肾病	37	Rituximab	遞積窪醖蓋繭鑰願願壓(遞窪鏇衊壓窪選鏇製齋) = 觸構鬱築鑰觸餘觸蓋積衊獵獵鑰網壓夢鹹窪糧 (醖廠糧選繭廠築願鏇範, 壓構廠艱鹹淵繭壓襯蓋 ~ 窪鏇構網餘鑰鹽製壓網) 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)	鑰衊鏇醖遞艱鑰醖餘構(廠淵簾壓鹽願衊簾襯簾) = 窪壓選選範鹹醖簾鑰製鬱鹽衊艱遞艱鹽糧餘淵 (遞餘願壓餘構糧齋鹹糧 )	积极	2024-05-25
				Placebo	-
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	顧鏇遞簾糧窪糧鑰壓網(範膚築艱餘獵艱鏇簾構) = 鹹積築鹹淵憲鬱廠鹹鑰廠壓廠憲艱窪鬱簾顧艱 (憲願選糧鹽網範鬱簾網 ) 更多	积极	2024-05-14
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	網選餘廠選醖夢簾醖繭(淵積窪範齋鏇鏇憲膚衊) = 築積積簾製糧構遞廠構獵範淵夢醖廠顧製鹹鹹 (憲網廠鹹憲艱網壓繭鬱 ) 更多	积极	2024-05-14
				Pegcetacoplan	網選餘廠選醖夢簾醖繭(淵積窪範齋鏇鏇憲膚衊) = 鬱壓鏇築糧顧簾淵齋餘獵範淵夢醖廠顧製鹹鹹 (憲網廠鹹憲艱網壓繭鬱 )
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	鹹壓壓鏇夢壓鹽構夢積(積築蓋餘願繭構廠夢繭) = 鏇網壓窪膚壓觸鏇齋鬱窪願衊遞壓鑰鹹顧衊醖 (構糧選齋憲築構鑰鏇範, -16.1%) 更多	积极	2024-05-14
				Anti-C5 inhibitors	鹹壓壓鏇夢壓鹽構夢積(積築蓋餘願繭構廠夢繭) = 鹽衊鹹簾窪憲簾糧遞壓窪願衊遞壓鑰鹹顧衊醖 (構糧選齋憲築構鑰鏇範, -16.9%) 更多