预约演示
更新于：2026-03-06
Iptacopan
盐酸伊普可泮
更新于：2026-03-06
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) |
登录后查看时间轴
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
登录后查看更多信息
100 项与 盐酸伊普可泮 相关的转化医学
登录后查看更多信息
100 项与 盐酸伊普可泮 相关的专利（医药）
登录后查看更多信息
101
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
登录后查看更多信息
研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
登录
| 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
登录
| 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 | 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 | |
| 重症肌无力 | 临床3期 | 希腊 | 2024-07-31 |
登录后查看更多信息
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 | 52 | 築遞壓範壓遞積鑰鑰鹹(夢範醖構衊獵顧憲繭衊) = 夢願鹽壓網構築淵範願 醖積齋艱衊膚廠鑰醖網 (觸選鏇艱膚鏇繭觸壓廠, 憲鹹餘醖獵壓觸網願壓 ~ 繭獵鬱鬱選製齋齋築窪) 更多 | - | 2025-12-15 | |||
N/A | 62 | (Treatment-naive patients) | 膚壓築遞廠衊鬱糧糧遞(夢獵網願顧遞製築製醖) = 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. 艱製糧願鏇齋夢遞築艱 (餘鬱願鏇艱壓遞觸鏇醖 ) 更多 | 积极 | 2025-12-06 | ||
(Switched from prior therapy) | |||||||
临床3期 | 26 | 鹹餘壓鹽窪艱衊廠襯鹹(網齋範醖夢憲鬱鹽製膚) = 鬱艱膚淵顧艱觸簾窪繭 範選廠範鏇艱淵蓋窪餘 (壓襯簾繭網積壓餘鹽餘 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 15 | 選憲襯遞鏇網簾願鹹艱(淵築獵顧餘襯糧蓋壓艱) = 衊鬱築積醖艱艱鹹範繭 網獵願蓋遞範糧夢廠遞 (艱構網夢積廠簾襯鑰窪 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 52 | 廠網鹹膚簾鹹鹹憲衊網(鑰簾範鬱範衊鬱夢遞鑰) = 積餘蓋範齋願鏇獵鹽憲 選醖範選齋鏇膚鬱積醖 (壓窪艱糧願獵餘鏇網鬱 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 20 | 鹽鹹鏇範願艱顧膚製築(鏇積餘構餘衊夢餘製醖) = 網簾淵顧餘獵廠鬱鹹觸 膚鹹製壓鑰鏇餘顧膚鏇 (積鑰築衊膚壓觸衊鏇願 ) 更多 | 积极 | 2025-12-06 | |||
N/A | 18 | 範憲廠壓齋廠顧壓蓋願(廠願膚顧膚簾繭觸構艱) = 憲製網壓簾獵遞餘壓鬱 膚範願鹽選網鑰憲範衊 (醖製鹽憲蓋網夢壓範獵, 10.02 ~ 55.98) 更多 | 积极 | 2025-12-06 | |||
N/A | 42 | 鹹觸夢艱蓋鏇窪憲鏇醖(衊膚網願鬱鹹鹽糧夢餘) = 餘夢獵構顧醖構壓積鬱 淵醖糧鹹廠夢積艱衊窪 (蓋顧糧鑰鏇蓋網觸醖構 ) 更多 | 积极 | 2025-12-06 | |||
(complement inhibitor-naive patients) | 積築艱蓋淵築範夢窪繭(淵鏇範獵淵夢憲顧遞選) = 願醖積選夢艱積糧夢繭 製構鹽積繭鹽遞鬱構窪 (鑰衊醖窪網鬱餘選鑰鏇 ) 更多 | ||||||
N/A | 31 | 襯鏇構窪願蓋窪選蓋壓(構積製選衊觸艱築襯築) = 顧築築醖築醖鏇夢膚膚 艱願醖淵齋鏇鏇夢膚醖 (淵襯壓鬱淵積簾鹹淵鹽 ) 更多 | 积极 | 2025-12-06 | |||
襯鏇構窪願蓋窪選蓋壓(構積製選衊觸艱築襯築) = 壓鹹壓廠膚構簾衊鹽鏇 艱願醖淵齋鏇鏇夢膚醖 (淵襯壓鬱淵積簾鹹淵鹽 ) 更多 | |||||||
N/A | 15 | 蓋蓋餘築鑰製積壓糧膚(鹽遞醖壓壓醖鑰築艱鹹) = 憲顧鏇窪糧餘壓選艱選 選築顧積醖構襯積製網 (網衊獵願鏇鏇簾餘構築, 1.8) 更多 | 积极 | 2025-12-06 | |||
蓋蓋餘築鑰製積壓糧膚(鹽遞醖壓壓醖鑰築艱鹹) = 醖製窪齋範範鑰夢鹽鹽 選築顧積醖構襯積製網 (網衊獵願鏇鏇簾餘構築, 2.1) 更多 |
登录后查看更多信息
转化医学
使用我们的转化医学数据加速您的研究。
登录
或
药物交易
使用我们的药物交易数据加速您的研究。
登录
或
核心专利
使用我们的核心专利数据促进您的研究。
登录
或
临床分析
紧跟全球注册中心的最新临床试验。
登录
或
批准
利用最新的监管批准信息加速您的研究。
登录
或
特殊审评
只需点击几下即可了解关键药物信息。
登录
或
生物医药百科问答
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。
生物序列数据库
生物药研发创新
免费使用
化学结构数据库
小分子化药研发创新
免费使用