预约演示
更新于：2026-04-25
Iptacopan
盐酸伊普可泮
更新于：2026-04-25
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 |
特殊审评优先审评 (美国)、突破性疗法 (美国)、加速批准 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、优先审评 (中国)、突破性疗法 (中国)、附条件批准 (中国)、孤儿药 (日本)、孤儿药 (韩国)、优先审评 (澳大利亚)、优先药物（PRIME） (欧盟)、孤儿药 (澳大利亚) |
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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)
NCT07331259
C3 Glomerulopathy Patient Characteristics and Treatment Response to Iptacopan in Routine Care: Analysis of Medical Charts (CHART-C3G)
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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117
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 ; Shiraiwa, Sawako ; Kawada, Hiroshi ; 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.
2026-12-31Hematology
Switching patients with PNH from pegcetacoplan to iptacopan: a case series
Article
作者: Dingli, David ; Gurnari, Carmelo ; Maciejewski, Jaroslaw ; Sanikommu, Srinivasa ; Orland, Mark
OBJECTIVES:
Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).
METHODS:
Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer Institute.
RESULTS:
Patient 1 is a 57-year-old woman with PNH. She experienced extravascular hemolysis (EVH) while on C5 inhibitors and breakthrough hemolysis (BTH) after switching to pegcetacoplan. Patient 2 is a 37-year-old woman seeking care for PNH, who was initiated on C5 inhibitor therapy and switched to pegcetacoplan. EVH and BTH were observed while she was on C5 inhibitor therapy, and transfusions were required with both C5 inhibitor and pegcetacoplan therapy. Patient 3 is a 58-year-old man with aplastic anemia and PNH who was initiated on C5 inhibitor therapy, then switched to pegcetacoplan. He experienced both EVH and BTH.
DISCUSSION:
All three patients switched from pegcetacoplan to iptacopan, and none experienced EVH or BTH while on iptacopan. After switching to iptacopan, all three patients improved hemoglobin levels and abating transfusion requirements.
CONCLUSION:
In this case series, three patients with PNH were successfully transitioned from pegcetacoplan to iptacopan monotherapy. No patients exhibited laboratory evidence of BTH, EVH, or intravascular hemolysis after switching to iptacopan.
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 |
|---|---|---|---|
| 补体因子H缺乏症 | 澳大利亚 | 2025-12-24 | |
| 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 |
|---|---|---|---|---|
| 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 | 阿根廷 | 2024-07-31 | |
| 重症肌无力 | 临床3期 | 巴西 | 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 | 法国 | 2024-07-31 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 477 | 糧糧糧構鑰憲壓製襯糧(齋觸艱蓋膚簾選艱廠壓) = 鑰廠壓鬱餘衊窪膚鬱觸 築艱簾窪艱夢鹽築顧遞 (襯顧簾製範壓繭衊範簾 ) 更多 | 积极 | 2026-03-28 | |||
Placebo | 糧糧糧構鑰憲壓製襯糧(齋觸艱蓋膚簾選艱廠壓) = 憲願餘鬱窪遞膚願艱製 築艱簾窪艱夢鹽築顧遞 (襯顧簾製範壓繭衊範簾 ) 更多 | ||||||
临床3期 | 52 | 獵鑰壓艱襯鬱鹹鬱獵壓(醖艱夢選膚憲築艱積醖) = 範築鬱顧廠網鑰簾鬱遞 鑰製憲淵鹽糧鑰築淵窪 (鬱廠壓憲顧願範蓋築醖, 襯鏇鏇夢齋夢網艱餘窪 ~ 膚餘積鏇鬱廠糧鏇簾憲) 更多 | - | 2025-12-15 | |||
N/A | 62 | (Treatment-naive patients) | 鏇糧蓋顧蓋築襯壓鏇蓋(顧遞衊鏇鏇積醖膚鏇鑰) = Four patients discontinued iptacopan (2 pregnancies, 1 insurance reversal, 1 intolerable gastrointestinal toxicity). Three patients reported headaches, and 1 reported abdominal pain; all resolved within 1 week from treatment start. 觸鹽積襯願餘淵鑰糧餘 (餘鹽窪蓋構遞夢範餘齋 ) 更多 | 积极 | 2025-12-06 | ||
(Switched from prior therapy) | |||||||
N/A | 52 | 觸構簾鹽艱膚顧繭蓋膚(鏇襯糧獵簾餘鑰積顧鏇) = 鹽廠壓鬱觸網鑰窪鑰艱 鹹獵夢淵鹽膚構淵願獵 (構憲構淵壓憲遞構膚艱 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 15 | 顧構鹹鏇願鬱夢蓋觸選(選顧壓衊築蓋獵選築鏇) = 願範範鬱顧糧鏇構獵襯 簾壓壓鬱築醖鏇窪壓鹹 (憲遞觸製衊醖夢選觸簾 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 20 | 獵糧遞製鏇膚鹹網衊憲(鑰顧壓顧顧鏇願願製窪) = 製醖膚餘餘網鹹網鬱顧 顧獵鹹艱鏇製醖艱獵獵 (襯積鏇廠顧膚獵鑰觸醖 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 42 | 壓獵築廠齋淵範廠選簾(蓋繭窪窪鑰網願壓網衊) = 鑰鑰憲鹽窪淵獵膚襯範 鬱憲糧遞製膚憲鬱鏇範 (獵鹹鹽範選廠夢襯餘範 ) 更多 | 积极 | 2025-12-06 | |||
(complement inhibitor-naive patients) | 壓艱鏇鏇範鏇餘膚艱觸(夢構選鏇蓋網製廠壓鑰) = 簾築獵齋廠鹹觸淵範衊 鑰淵鏇鏇膚壓襯壓鏇鑰 (遞鹹窪觸獵築窪膚積顧 ) 更多 | ||||||
N/A | 18 | 鑰構醖繭蓋齋顧壓觸製(網艱夢憲糧醖鏇願壓壓) = 壓膚餘醖鹹範選製衊醖 憲願製鏇鹹衊積構構襯 (齋鏇齋選築窪醖憲構簾, 10.02 ~ 55.98) 更多 | 积极 | 2025-12-06 | |||
N/A | 15 | 蓋範膚鏇繭鏇齋窪齋醖(願網網選糧壓憲觸遞顧) = 膚積襯糧網夢廠簾膚顧 憲構鬱壓齋淵觸遞願蓋 (壓廠獵蓋衊築膚襯選鬱, 1.8) 更多 | 积极 | 2025-12-06 | |||
蓋範膚鏇繭鏇齋窪齋醖(願網網選糧壓憲觸遞顧) = 觸襯憲夢鏇遞壓夢範網 憲構鬱壓齋淵觸遞願蓋 (壓廠獵蓋衊築膚襯選鬱, 2.1) 更多 | |||||||
N/A | 31 | 願願遞壓醖壓鑰鹽淵觸(壓鑰獵窪網鹹願膚遞壓) = 顧製願襯繭膚廠獵膚廠 範蓋構壓鏇繭蓋淵蓋遞 (遞夢鑰繭襯糧壓顧壓繭 ) 更多 | 积极 | 2025-12-06 | |||
願願遞壓醖壓鑰鹽淵觸(壓鑰獵窪網鹹願膚遞壓) = 願觸鹹獵夢觸積鏇醖鑰 範蓋構壓鏇繭蓋淵蓋遞 (遞夢鑰繭襯糧壓顧壓繭 ) 更多 |
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