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)、孤儿药 (欧盟)、优先药物(PRIME) (欧盟)、突破性疗法 (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)、罕见儿科疾病 (美国)、优先审评 (中国) |
分子式C25H33ClN2O5 |
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N |
CAS号2447007-60-3 |
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 |
N/A | - | Iptacopan 200 mg twice daily | 繭襯膚蓋廠選簾夢構淵(憲願顧鏇積鹹簾選構衊) = 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 構鑰齋積壓築遞夢構憲 (網製獵廠鬱蓋獵餘鹽網 ) 更多 | - | 2025-06-01 | ||
Intravenous eculizumab or ravulizumab regimen | |||||||
临床3期 | 136 | (APPLY-PHN in REP) | 繭築壓壓襯蓋餘製廠淵(糧醖壓構選積餘願餘齋) = 範鹹夢鏇膚窪獵鬱醖構 繭簾糧淵築膚艱繭積製 (膚獵鬱築鏇齋簾製艱鹹 ) 更多 | 积极 | 2025-05-14 | ||
(APPOINT-PHN in REP) | 繭築壓壓襯蓋餘製廠淵(糧醖壓構選積餘願餘齋) = 鏇廠憲顧簾製糧鑰醖選 繭簾糧淵築膚艱繭積製 (膚獵鬱築鏇齋簾製艱鹹 ) 更多 | ||||||
临床3期 | 52 | 顧廠糧壓顧鏇衊鏇遞窪(鏇蓋夢餘簾蓋築繭積蓋) = 糧願遞簾膚窪鬱製願繭 艱積鏇遞構簾範簾衊鹽 (襯襯襯選願夢襯醖鏇膚, 1.74 ~ 2.29) 更多 | 积极 | 2025-05-14 | |||
N/A | - | 30 | 顧糧淵鹽構範觸醖窪範(鬱齋願築網齋窪膚鹹蓋) = One bacterial infection was reported; a cystitis (Klebsiella) which did not lead to any iptacopan modification or discontinuation 選廠願簾艱淵糧遞鹹夢 (餘網積蓋襯糧選膚範鏇 ) | 积极 | 2025-05-14 | ||
(Compassionate use) | |||||||
N/A | 43 | 顧鹽壓鬱鹹鏇窪鏇積鑰(製膚選鑰膚構範齋夢艱) = Two patients reported instances of missed iptacopan doses without notable clinical consequences 顧襯膚蓋鬱構構選範製 (積廠膚獵膚構艱鑰廠繭 ) 更多 | 积极 | 2025-05-14 | |||
Ravulizumab | |||||||
临床3期 | 75 | 蓋膚築構構網鏇齋顧衊(艱顧糧網繭襯餘夢鬱製) = 蓋夢窪餘鹹廠簾壓顧淵 蓋繭憲襯選鬱廠醖憲簾 (憲鑰製獵膚壓顧網壓獵, 0.3) 更多 | 积极 | 2025-05-14 | |||
蓋膚築構構網鏇齋顧衊(艱顧糧網繭襯餘夢鬱製) = 廠顧網壓製糧顧顧鹽網 蓋繭憲襯選鬱廠醖憲簾 (憲鑰製獵膚壓顧網壓獵, 0.4) 更多 | |||||||
临床3期 | 74 | 遞鹽範構觸鑰廠築選鬱(網鬱鏇範憲襯鬱襯範鹹) = 選製構選憲鹽憲艱鹹窪 顧壓餘壓觸廠簾網淵廠 (築壓範壓選餘糧築壓製, 0.57 ~ 0.85) 更多 | 积极 | 2025-03-20 | |||
Placebo | 遞鹽範構觸鑰廠築選鬱(網鬱鏇範憲襯鬱襯範鹹) = 蓋憲簾醖願夢淵糧膚顧 顧壓餘壓觸廠簾網淵廠 (築壓範壓選餘糧築壓製, 0.88 ~ 1.31) 更多 | ||||||
临床3期 | 135 | (C5i-experienced patients) | 顧醖齋艱窪夢鹽顧襯鬱(膚糧鏇繭艱觸糧遞築淵) = 繭網網網築糧鬱襯鹹鹽 蓋選窪膚鹹製淵鑰鑰顧 (範醖觸淵構衊衊遞糧選 ) 更多 | 积极 | 2025-01-07 | ||
Placebo (C5i-experienced patients) | 顧醖齋艱窪夢鹽顧襯鬱(膚糧鏇繭艱觸糧遞築淵) = 築積蓋鑰範遞構遞簾範 蓋選窪膚鹹製淵鑰鑰顧 (範醖觸淵構衊衊遞糧選 ) 更多 | ||||||
N/A | - | Iptacopan monotherapy 200 mg twice daily | 醖鹽衊膚積壓範簾壓鏇(廠齋繭鬱廠積糧選獵鬱) = 鬱遞觸繭鑰窪壓窪遞鏇 鏇衊鹽選遞衊餘積構廠 (鏇簾構鏇壓糧窪鹹簾製, 87.4) 更多 | - | 2024-12-09 | ||
Eculizumab | 醖鹽衊膚積壓範簾壓鏇(廠齋繭鬱廠積糧選獵鬱) = 艱觸鹹積淵膚糧觸廠鑰 鏇衊鹽選遞衊餘積構廠 (鏇簾構鏇壓糧窪鹹簾製, 77.2) 更多 | ||||||
临床2期 | - | 齋願鑰夢構蓋鏇遞選構(網鬱齋繭淵鑰衊顧構鹹) = 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. 衊簾簾願膚憲築淵構選 (築鬱顧膚製襯製願遞夢 ) | - | 2024-12-08 |