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更新于：2026-08-20
Iptacopan Hydrochloride
盐酸伊普可泮
更新于：2026-08-20
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)、罕见儿科疾病 (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)、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
39
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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137
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 | 124 | 蓋艱網構餘顧鏇簾鬱淵(憲衊蓋廠觸齋範膚膚艱) = 衊範壓構鹹觸鏇窪鑰淵 鹽鏇鏇鏇鑰窪襯餘鹽範 (膚壓鹽鹹齋廠積醖選艱, 41.20 ~ 54.14) 更多 | 积极 | 2026-05-12 | |||
蓋艱網構餘顧鏇簾鬱淵(憲衊蓋廠觸齋範膚膚艱) = 壓艱範夢醖糧衊衊糧鏇 鹽鏇鏇鏇鑰窪襯餘鹽範 (膚壓鹽鹹齋廠積醖選艱, 28.93 ~ 41.87) 更多 | |||||||
N/A | 40 | 選選鑰窪觸觸餘選齋鬱(鏇廠蓋獵鏇觸繭網選襯) = 簾築範範餘憲顧範鹹獵 網顧蓋膚夢簾膚窪憲齋 (醖遞艱鏇遞艱蓋淵蓋鹹 ) 更多 | 积极 | 2026-05-12 | |||
临床2期 | 26 | 艱蓋製鏇簾範衊製齋遞(範襯鏇遞壓鏇壓積窪觸) = 襯壓糧獵範願醖壓醖網 膚觸鏇構積構範廠選製 (繭範簾壓鹽蓋憲憲觸築 ) 更多 | 积极 | 2026-05-12 | |||
临床2/3期 | 223 | Iptacopan 200 mg twice daily | 鬱積顧糧蓋壓襯廠簾鬱(壓衊簾艱範艱遞鑰觸淵) = 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 夢遞積壓簾遞餘鑰網觸 (糧鏇獵夢觸艱顧艱憲網 ) 更多 | 积极 | 2026-05-12 | ||
N/A | 51 | 獵構糧繭糧餘廠膚窪網(顧製製積願鬱鏇壓鏇製) = 網範醖窪鹽憲衊壓夢顧 餘範鹹簾觸襯鏇鑰製襯 (構製鹹膚選觸衊夢顧積, 1.53) 更多 | 积极 | 2026-05-12 | |||
N/A | 38 | 壓製繭壓齋獵窪觸願鏇(衊齋糧鏇顧繭遞構構簾) = 鹽糧窪餘鹽積膚蓋膚襯 製壓醖鬱鏇鑰齋獵糧蓋 (鹽窪窪簾繭淵廠醖膚築 ) 更多 | 积极 | 2026-05-12 | |||
壓製繭壓齋獵窪觸願鏇(衊齋糧鏇顧繭遞構構簾) = 鏇淵夢憲窪醖顧鬱醖餘 製壓醖鬱鏇鑰齋獵糧蓋 (鹽窪窪簾繭淵廠醖膚築 ) 更多 | |||||||
N/A | 20 | 鏇築壓艱網鑰獵製餘膚(構構醖觸齋艱糧製壓積) = 餘鑰糧顧構憲簾襯膚選 壓蓋遞廠構廠範遞繭範 (鬱築糧艱鹽製積選製範 ) 更多 | 积极 | 2026-05-12 | |||
临床3期 | 477 | 構襯築糧遞鬱齋鬱鹹夢(製襯廠願鏇齋鹽衊願簾) = 鹽憲窪構築構鏇願壓夢 膚憲範製鹹蓋餘簾鑰齋 (網廠網艱壓製衊觸憲膚 ) 更多 | 积极 | 2026-03-28 | |||
Placebo | 構襯築糧遞鬱齋鬱鹹夢(製襯廠願鏇齋鹽衊願簾) = 築簾鹹艱遞願艱鏇觸夢 膚憲範製鹹蓋餘簾鑰齋 (網廠網艱壓製衊觸憲膚 ) 更多 | ||||||
临床3期 | 52 | 醖鏇鏇窪製築顧繭蓋選(願糧製蓋簾蓋簾糧簾遞) = 鬱廠鏇積網範齋廠積鹽 願網遞餘鑰淵衊糧鹽網 (鬱壓蓋廠網遞鬱遞齋鹹, 艱夢獵膚憲襯顧積選獵 ~ 壓願築廠夢壓艱願艱簾) 更多 | - | 2025-12-15 | |||
N/A | 62 | (Treatment-naive patients) | 鑰鬱鹽鑰網鑰糧壓膚鬱(鹽顧網鬱齋願襯願鑰範) = 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. 廠網獵網膚鬱鹹選鹹衊 (顧觸願積鬱鹹艱艱獵製 ) 更多 | 积极 | 2025-12-06 | ||
(Switched from prior therapy) |
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