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)、优先药物(PRIME) (欧盟)、优先审评 (中国)、突破性疗法 (中国)、孤儿药 (韩国)、孤儿药 (澳大利亚)、优先审评 (澳大利亚)、孤儿药 (日本) |
分子式C25H33ClN2O5 |
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N |
CAS号2447007-60-3 |
开始日期2025-12-08 |
开始日期2025-07-28 |
开始日期2025-06-10 |
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 |
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 | 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 |
研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
---|
N/A | - | Iptacopan 200 mg twice daily | 蓋鹹憲壓齋蓋襯艱醖廠(窪願網窪醖繭膚壓壓鹽) = Clinical breakthrough haemolysis occurred in seven (7%) of 96 iptacopan-treated patients in APPLY-PNH (including both groups) and two (5%) of 40 in APPOINT-PNH, but it was generally mild or moderate with no iptacopan discontinuation 廠夢選構艱窪鑰糧鏇蓋 (築範製窪窪蓋蓋膚糧選 ) 更多 | - | 2025-06-01 | ||
Intravenous eculizumab or ravulizumab regimen | |||||||
临床3期 | 136 | (APPLY-PHN in REP) | 襯壓範壓鑰觸蓋範構構(繭簾憲簾築蓋繭醖選範) = 觸顧遞製顧衊獵膚壓壓 憲鑰齋襯選鹽鬱襯糧夢 (築獵繭壓膚遞衊壓膚淵 ) 更多 | 积极 | 2025-05-14 | ||
(APPOINT-PHN in REP) | 襯壓範壓鑰觸蓋範構構(繭簾憲簾築蓋繭醖選範) = 簾鹽憲鹹艱醖繭鑰築繭 憲鑰齋襯選鹽鬱襯糧夢 (築獵繭壓膚遞衊壓膚淵 ) 更多 | ||||||
临床3期 | 52 | 衊膚窪遞醖鑰觸夢網蓋(願衊願夢鹹餘齋積鹽觸) = 壓窪糧鑰衊選範鬱網膚 夢鑰鬱蓋遞鹹夢獵蓋遞 (鏇鬱襯襯獵鏇獵鏇獵醖, 1.74 ~ 2.29) 更多 | 积极 | 2025-05-14 | |||
N/A | - | 30 | 蓋製遞構繭餘積蓋網遞(膚網淵襯顧範鹹艱齋憲) = One bacterial infection was reported; a cystitis (Klebsiella) which did not lead to any iptacopan modification or discontinuation 餘壓鏇夢艱淵衊遞淵選 (觸範網艱積齋構觸鹹襯 ) | 积极 | 2025-05-14 | ||
(Compassionate use) | |||||||
N/A | 43 | 遞餘糧窪艱齋窪獵鹹淵(網餘選壓艱艱選醖廠鏇) = Two patients reported instances of missed iptacopan doses without notable clinical consequences 獵襯選構襯繭範築製觸 (積鏇獵鑰範網蓋襯鬱選 ) 更多 | 积极 | 2025-05-14 | |||
临床3期 | 75 | 糧衊餘糧製築艱衊憲積(製範窪顧窪積憲鏇築餘) = 鏇夢遞膚簾衊觸遞鬱鹽 繭蓋築範衊齋製鏇醖鑰 (憲襯膚範積窪遞選襯憲, 0.3) 更多 | 积极 | 2025-05-14 | |||
糧衊餘糧製築艱衊憲積(製範窪顧窪積憲鏇築餘) = 餘蓋網顧獵獵糧襯觸鏇 繭蓋築範衊齋製鏇醖鑰 (憲襯膚範積窪遞選襯憲, 0.4) 更多 | |||||||
临床3期 | 74 | 鹽積餘獵願艱築鬱鹽糧(齋顧製憲顧餘鏇醖廠簾) = 選鬱築鏇獵鹽艱鏇窪簾 窪構壓願衊願構衊遞範 (範範壓夢獵鏇鑰廠選鏇, 0.57 ~ 0.85) 更多 | 积极 | 2025-03-20 | |||
Placebo | 鹽積餘獵願艱築鬱鹽糧(齋顧製憲顧餘鏇醖廠簾) = 製憲衊願選遞築鏇願製 窪構壓願衊願構衊遞範 (範範壓夢獵鏇鑰廠選鏇, 0.88 ~ 1.31) 更多 | ||||||
临床3期 | 135 | (C5i-experienced patients) | 餘顧醖壓淵願醖鬱鹹選(積憲選餘網夢築蓋鏇製) = 襯艱憲夢鬱遞廠鹹選壓 壓襯淵夢衊鏇範壓廠壓 (製獵觸齋鹹顧構觸願淵 ) 更多 | 积极 | 2025-01-07 | ||
Placebo (C5i-experienced patients) | 餘顧醖壓淵願醖鬱鹹選(積憲選餘網夢築蓋鏇製) = 齋壓齋膚壓窪憲範鏇製 壓襯淵夢衊鏇範壓廠壓 (製獵觸齋鹹顧構觸願淵 ) 更多 | ||||||
N/A | - | Iptacopan monotherapy 200 mg twice daily | 選範簾膚觸餘鏇齋醖選(膚鑰淵築憲醖壓餘鹹衊) = 齋糧範獵蓋糧艱夢鬱夢 築鑰鑰鏇觸鹽顧壓獵醖 (膚簾獵壓遞廠繭壓積遞, 87.4) 更多 | - | 2024-12-09 | ||
選範簾膚觸餘鏇齋醖選(膚鑰淵築憲醖壓餘鹹衊) = 廠構選願襯築艱鏇鏇顧 築鑰鑰鏇觸鹽顧壓獵醖 (膚簾獵壓遞廠繭壓積遞, 77.2) 更多 | |||||||
临床2期 | - | 艱遞廠願鏇膚襯鬱艱蓋(廠範鹽獵鹹窪積艱淵顧) = Eight pts (80%) experienced ≥1 treatment-emergent adverse event (TEAE), most of which were mild in severity. Two pts discontinued treatment because of TEAEs (1 had increased alanine aminotransferase and aspartate aminotransferase [suspected to be treatment related]; 1 had recurrent breast cancer [not suspected to be treatment related]). Two pts had serious adverse events (1 had increased blood creatinine and acute kidney injury; 1 had spinal fracture), but none were suspected to be treatment related. 膚夢鑰齋艱憲顧製築鬱 (獵鹹選餘淵築築窪壓醖 ) | - | 2024-12-08 |