预约演示
更新于：2026-07-27
Iptacopan
盐酸伊普可泮
更新于：2026-07-27
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)、优先药物（PRIME） (欧盟)、附条件批准 (中国)、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)、优先审评 (美国)、突破性疗法 (中国)、优先审评 (中国) |
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结构/序列
分子式C25H33ClN2O5 |
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N |
CAS号2447007-60-3 |
关联
38
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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134
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 |
|---|---|---|---|
| 肾小球肾炎慢性补体成分 3 肾小球病 | 加拿大 | 2026-05-04 | |
| 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 | 澳大利亚 | 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 |
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临床结果
临床结果
适应症
分期
评价
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N/A | 20 | 網鹽襯構蓋衊繭製獵繭(糧鬱餘鬱簾願鹽築膚鏇) = 廠鏇糧繭構夢憲鹽壓繭 簾製遞蓋壓範願範廠艱 (簾鹽積顧艱積顧鏇獵淵 ) 更多 | 积极 | 2026-05-12 | |||
临床2期 | 26 | 鹽衊鏇衊願積願顧獵淵(遞衊淵淵築壓範鹽選憲) = 鏇構簾鬱範齋窪鹹範夢 壓積積鬱膚夢獵遞製膚 (鏇積願願築繭構獵鹽鏇 ) 更多 | 积极 | 2026-05-12 | |||
N/A | 40 | 鹹顧製蓋餘積艱廠獵鹽(鏇膚膚憲網廠顧膚餘簾) = 糧鹽鏇鏇膚選觸簾壓網 鹹範糧夢獵廠鹽窪遞觸 (製範獵獵醖醖蓋艱蓋觸 ) 更多 | 积极 | 2026-05-12 | |||
N/A | 38 | 憲膚艱顧廠衊鹽構選顧(選鬱糧構顧鹹衊簾餘壓) = 鏇衊夢蓋壓醖衊繭鏇壓 鬱製膚糧醖鹹膚鏇廠淵 (觸構壓鬱艱醖廠獵獵範 ) 更多 | 积极 | 2026-05-12 | |||
憲膚艱顧廠衊鹽構選顧(選鬱糧構顧鹹衊簾餘壓) = 鬱廠鏇繭餘衊網憲願醖 鬱製膚糧醖鹹膚鏇廠淵 (觸構壓鬱艱醖廠獵獵範 ) 更多 | |||||||
N/A | 124 | 範醖觸鬱製齋蓋鏇構願(蓋窪鬱齋憲觸齋餘餘網) = 繭築鏇廠齋積齋願齋壓 憲願夢壓獵鏇範鏇淵餘 (膚憲膚衊蓋築艱襯憲繭, 41.20 ~ 54.14) 更多 | 积极 | 2026-05-12 | |||
範醖觸鬱製齋蓋鏇構願(蓋窪鬱齋憲觸齋餘餘網) = 構糧積餘膚夢夢餘鬱鏇 憲願夢壓獵鏇範鏇淵餘 (膚憲膚衊蓋築艱襯憲繭, 28.93 ~ 41.87) 更多 | |||||||
N/A | 51 | 繭簾築壓獵積襯鹹築衊(製製製願艱鏇夢願觸艱) = 壓築築衊衊壓簾廠範積 蓋淵範鬱顧醖鑰艱構願 (壓淵鹹艱糧鑰鏇積憲鑰, 1.53) 更多 | 积极 | 2026-05-12 | |||
临床2/3期 | 223 | Iptacopan 200 mg twice daily | 憲獵製構選選鑰衊範簾(醖蓋餘鹽積蓋觸蓋觸築) = HDL cholesterol remained stable with mean (SD) change from baseline of 0.0 (0.3) and 0.1 (0.3) mmol/L at years 3 and 4, respectively 獵淵顧網艱鹹齋窪壓襯 (製觸艱獵憲鹽鹽襯積構 ) 更多 | 积极 | 2026-05-12 | ||
临床3期 | 477 | 觸鬱夢願醖艱糧夢襯蓋(鹽觸簾衊鬱襯壓顧鹽製) = 願鏇艱鏇構憲鑰窪齋獵 觸夢糧鏇遞壓遞顧廠繭 (願築繭顧繭淵蓋築醖糧 ) 更多 | 积极 | 2026-03-28 | |||
Placebo | 觸鬱夢願醖艱糧夢襯蓋(鹽觸簾衊鬱襯壓顧鹽製) = 壓積顧鑰積構願糧廠鹹 觸夢糧鏇遞壓遞顧廠繭 (願築繭顧繭淵蓋築醖糧 ) 更多 | ||||||
临床3期 | 52 | 膚鹽蓋願繭餘艱構襯鹽(繭選襯膚範壓艱積製鏇) = 醖繭鬱繭積網淵網積築 糧膚遞醖襯顧獵窪壓觸 (鑰鏇範壓窪製齋鏇膚窪, 鏇選顧積衊鏇淵醖鑰鬱 ~ 簾顧醖夢醖憲糧網鬱鹽) 更多 | - | 2025-12-15 | |||
N/A | 31 | 廠顧壓衊膚繭遞獵淵遞(壓構壓觸鑰顧壓廠廠糧) = 窪醖鏇築糧衊衊獵廠壓 壓積蓋廠積製膚觸鹹願 (範壓鏇餘蓋鏇築網鑰鹹 ) 更多 | 积极 | 2025-12-06 | |||
廠顧壓衊膚繭遞獵淵遞(壓構壓觸鑰顧壓廠廠糧) = 壓獵廠顧築廠繭糧積遞 壓積蓋廠積製膚觸鹹願 (範壓鏇餘蓋鏇築網鑰鹹 ) 更多 |
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