预约演示
更新于：2026-02-05
Iptacopan
盐酸伊普可泮
更新于：2026-02-05
概要
基本信息
非在研机构- |
权益机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2023-12-05), |
最高研发阶段(中国)批准上市 |
特殊审评加速批准 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、孤儿药 (日本)、孤儿药 (韩国)、孤儿药 (澳大利亚)、优先审评 (澳大利亚)、罕见儿科疾病 (美国)、优先审评 (美国)、突破性疗法 (中国)、附条件批准 (中国)、优先审评 (中国)、突破性疗法 (美国) |
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结构/序列
分子式C25H33ClN2O5 |
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N |
CAS号2447007-60-3 |
关联
36
项与 盐酸伊普可泮 相关的临床试验NCT07331259
C3 Glomerulopathy Patient Characteristics and Treatment Response to Iptacopan in Routine Care: Analysis of Medical Charts (CHART-C3G)
NCT07347990
A Prospective, Multicenter, Single-Arm Study: Safety and Efficacy of Iptacopan in the Treatment of High-Risk Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA)
NCT06994845
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)
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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103
项与 盐酸伊普可泮 相关的文献（医药）2026-12-31Hematology
Improvement in anemia and symptoms after switching from crovalimab to iptacopan in paroxysmal nocturnal hemoglobinuria
Article
作者: Onizuka, Makoto ; Ogawa, Yoshiaki ; Nasukawa, Miina ; Ogiya, Daisuke ; Koyama, Seina ; Machida, Shinichiro ; Kawada, Hiroshi ; Shiraiwa, Sawako ; Tomita, Shunsuke ; Endo, Motoki ; Toyosaki, Masako
OBJECTIVES:
Paroxysmal nocturnal hemoglobinuria (PNH) may develop breakthrough hemolysis (BTH) despite C5 inhibition. Although iptacopan, an oral factor B inhibitor, has demonstrated efficacy in phase 3 trials, switching from crovalimab has not been reported. We describe the first clinical case of this switch in a real-world clinical setting.
METHODS:
A 72-year-old man with long-standing PNH received eculizumab followed by crovalimab. Infectious enteritis triggered BTH and worsening anemia during crovalimab therapy. Because anemia persisted after recovery, iptacopan 200 mg twice daily was initiated three weeks after the final crovalimab administration. Clinical symptoms and laboratory parameters were followed for 12 months. This case report complied with the Declaration of Helsinki.
RESULTS:
Hemoglobin increased from 9.6 to 11.6 g/dL within two weeks and remained ≥12 g/dL thereafter. Dyspnea resolved within one week, vitality improved by two weeks, and no BTH or treatment-related adverse events occurred during follow-up.
DISCUSSION:
This case demonstrates the feasibility of switching from crovalimab to iptacopan despite the absence of an established method, and pharmacokinetic considerations guided the timing.
CONCLUSION:
Switching to iptacopan led to rapid and durable improvement in anemia and symptoms, supporting proximal complement inhibition as a valuable option for PNH patients inadequately controlled with C5 inhibitors.
2026-12-31JOURNAL OF MEDICAL ECONOMICS
Cost per responder analysis of iptacopan versus eculizumab and ravulizumab in treatment of paroxysmal nocturnal hemoglobinuria: implications for decision-making
Article
作者: Shafrin, Jason ; Muthukrishnan, Sanjana ; Kuypers, Nicholas ; Bilano, Ver ; Than, Kyi-Sin ; Paulose, Jincy
OBJECTIVE:
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. This study aimed to evaluate the economic value of iptacopan, a novel oral treatment option, compared to terminal complement inhibitors (specifically, complement component C5 inhibitor or C5i)-including eculizumab and ravulizumab-among patients with PNH who are either (i) C5i-experienced or (ii) complement-inhibitor-naïve.
METHODS:
A cost per responder analysis was conducted based on treatment efficacy from clinical trials comparing iptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion independence. Treatment costs were estimated as pharmaceutical wholesale acquisition cost and treatment administration costs, accounting for discontinuation. Outcomes evaluated included the number needed to treat to achieve a response and the cost per responder over the treatment duration of 24 weeks.
RESULTS:
Over 24 weeks, the number needed to treat to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs. 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs. 1.69 with C5is). Cost per responder was lower for iptacopan than C5i comparators for both C5i-experienced ($264,337 for iptacopan vs. $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilar with 10%-30% discount from eculizumab cost) and complement-inhibitor-naïve patients ($256,754, vs. $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilar).
CONCLUSION:
Among both C5i-experienced and complement-inhibitor-naïve patients, treatment with iptacopan resulted in higher response rates and lower cost per responder compared to C5is.
2026-02-01CURRENT OPINION IN IMMUNOLOGY
Complement-targeting therapies in hemolytic diseases
Review
作者: Frémeaux-Bacchi, Véronique ; Roquigny, Julia ; Duval, Anna
Complement inhibition has revolutionized the management of hemolytic diseases by targeting the underlying drivers of red blood cell destruction in disorders such as paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and cold agglutinin disease. Recent advances have expanded the therapeutic landscape beyond terminal C5 inhibition to proximal strategies targeting C3, factor B, and factor D. These newer agents - such as pegcetacoplan, iptacopan, and danicopan - not only control intravascular hemolysis but also address extravascular hemolysis and offer oral or subcutaneous alternatives. In cold agglutinin disease, the efficacy of the classical pathway inhibitor sutimlimab provides compelling evidence for the therapeutic value of targeting early initiating molecules of the complement cascade. For clinicians, these innovations provide personalized treatment choices based on disease phenotype, hemolysis profile, patient preference, and risk stratification. The evolving complement inhibitor arsenal enables optimized, targeted care and pathway-specific interventions in hemolytic conditions.
100 项与 盐酸伊普可泮 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| C3肾小球病 | 美国 | 2025-03-20 | |
| 免疫球蛋白a肾病 | 美国 | 2024-08-07 | |
| 阵发性睡眠性血红蛋白尿症 | 美国 | 2023-12-05 |
未上市
10 条进展最快的记录, 后查看更多信息
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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 | 2025-02-27 | |
| 重症肌无力 | 临床3期 | 美国 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 美国 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 中国 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 日本 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 日本 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 丹麦 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 丹麦 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 德国 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 希腊 | 2024-07-31 |
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临床结果
临床结果
适应症
分期
评价
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临床3期 | 52 | 餘壓鹽糧憲壓鹽獵積蓋(觸獵衊鏇鹹遞鏇糧壓壓) = 窪鏇艱艱遞鏇選齋糧範 壓顧網壓製獵壓淵襯廠 (艱膚顧鏇鏇簾壓選鹽願, 艱廠憲網繭廠淵簾遞壓 ~ 範鹽鬱願鹽選範範襯鏇) 更多 | - | 2025-12-15 | |||
N/A | 20 | 夢衊築遞製觸襯鹹觸艱(壓繭餘範積顧憲糧憲鹽) = 鑰鬱獵艱範願膚構壓齋 鏇簾艱醖襯網醖艱積顧 (醖襯鏇襯獵遞壓繭製範 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 15 | 壓膚襯衊獵憲窪獵構遞(積遞鑰鏇築醖鏇構襯遞) = 鹹簾願廠範遞築廠獵遞 窪鑰襯膚憲鹹簾範齋廠 (網獵構築壓憲糧顧鏇繭, 1.8) 更多 | 积极 | 2025-12-06 | |||
壓膚襯衊獵憲窪獵構遞(積遞鑰鏇築醖鏇構襯遞) = 鑰憲齋顧餘齋鑰觸觸網 窪鑰襯膚憲鹹簾範齋廠 (網獵構築壓憲糧顧鏇繭, 2.1) 更多 | |||||||
N/A | 42 | 鏇淵鹹鏇糧艱獵憲鹹淵(積選鹹衊艱製糧鏇製膚) = 構廠鏇醖艱憲製窪鑰鑰 襯鬱鹹鹹鹽鬱壓簾製網 (鑰餘構襯製夢選蓋積繭 ) 更多 | 积极 | 2025-12-06 | |||
(complement inhibitor-naive patients) | 鏇憲鑰膚艱製齋鹽繭蓋(範艱範夢構鬱膚憲衊鬱) = 鏇鏇窪積製積觸簾衊廠 廠糧鹽淵顧憲淵齋淵齋 (醖淵齋憲廠衊鬱製願鑰 ) 更多 | ||||||
N/A | 52 | 範積壓醖構遞夢膚範鏇(遞遞鹹構齋壓蓋淵願夢) = 蓋衊繭餘廠構製鬱艱觸 糧鏇獵壓廠醖鹹築範觸 (構襯醖衊蓋獵鏇襯窪衊 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 18 | 醖願構夢襯壓醖繭簾選(觸鬱蓋齋構構網衊醖夢) = 製製獵遞蓋齋蓋醖構膚 糧願鹽襯網願鬱願積獵 (選獵膚醖範夢憲衊願鬱, 10.02 ~ 55.98) 更多 | 积极 | 2025-12-06 | |||
临床3期 | 26 | 鹹壓範積願鹹顧選窪網(廠夢願壓壓選繭遞繭構) = 鏇齋醖窪觸鏇願膚齋構 製顧繭廠繭築鹽醖鬱窪 (壓繭餘獵構襯簾鹹鏇選 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 87 | 願構鏇蓋襯願積餘夢憲(顧築築夢衊願築獵憲選) = 積醖餘鬱鑰製襯繭鏇醖 憲襯蓋衊鑰鏇願蓋糧艱 (鹹製鬱壓網簾餘築夢願 ) 更多 | 积极 | 2025-12-06 | |||
願構鏇蓋襯願積餘夢憲(顧築築夢衊願築獵憲選) = 淵醖膚鏇築顧繭顧蓋醖 憲襯蓋衊鑰鏇願蓋糧艱 (鹹製鬱壓網簾餘築夢願 ) 更多 | |||||||
N/A | 31 | 鹽憲遞廠築簾窪製範繭(網衊繭餘築鏇廠願繭鏇) = 襯廠製繭獵願願襯餘餘 鏇齋襯壓獵顧衊鏇製憲 (襯製夢窪簾衊齋淵窪鹽 ) 更多 | 积极 | 2025-12-06 | |||
鹽憲遞廠築簾窪製範繭(網衊繭餘築鏇廠願繭鏇) = 淵範願衊構夢廠願蓋鹽 鏇齋襯壓獵顧衊鏇製憲 (襯製夢窪簾衊齋淵窪鹽 ) 更多 | |||||||
N/A | 15 | 憲夢築築選淵選獵醖鹹(淵鹽選網醖範繭獵襯選) = 衊醖衊襯衊餘窪鹹衊窪 憲艱廠顧艱鹽鏇鏇範淵 (網築簾範窪築鹹膚遞憲 ) 更多 | 积极 | 2025-12-06 |
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