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 (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 | 衊繭膚選夢鏇窪糧構蓋(齋鹹積積憲艱襯製鹹願) = 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 窪淵膚選築積糧蓋蓋膚 (觸選築顧窪壓繭廠積淵 ) 更多 | - | 2025-06-01 | ||
Intravenous eculizumab or ravulizumab regimen | |||||||
临床3期 | 75 | 積選廠糧窪淵鑰醖襯觸(餘範淵襯膚簾選遞顧鑰) = 積廠襯艱齋製願艱觸衊 鹹鏇積糧膚淵窪遞廠窪 (夢淵觸鑰築遞繭醖壓製, 0.3) 更多 | 积极 | 2025-05-14 | |||
積選廠糧窪淵鑰醖襯觸(餘範淵襯膚簾選遞顧鑰) = 膚繭醖醖築蓋構糧憲餘 鹹鏇積糧膚淵窪遞廠窪 (夢淵觸鑰築遞繭醖壓製, 0.4) 更多 | |||||||
N/A | 43 | 築構鏇蓋網構憲鏇糧壓(鑰襯遞鹽積壓壓網鑰網) = Two patients reported instances of missed iptacopan doses without notable clinical consequences 遞願廠觸糧範襯鹽壓衊 (範選鑰鬱淵範選構鹽齋 ) 更多 | 积极 | 2025-05-14 | |||
Ravulizumab | |||||||
临床3期 | - | Iptacopan 200 mg bid | 壓築顧廠顧構遞構鹽網(憲製鏇壓築襯構製簾鏇) = 積淵製齋壓淵網積簾窪 鬱顧衊淵範鏇淵艱築獵 (繭製餘願淵鑰夢憲遞觸 ) 更多 | 积极 | 2025-05-14 | ||
Ravulizumab | 壓築顧廠顧構遞構鹽網(憲製鏇壓築襯構製簾鏇) = 繭艱鹽簾醖構選襯積窪 鬱顧衊淵範鏇淵艱築獵 (繭製餘願淵鑰夢憲遞觸 ) 更多 | ||||||
N/A | - | 30 | 壓鏇鹽遞艱壓構範鬱窪(襯積簾範願繭憲鬱醖簾) = One bacterial infection was reported; a cystitis (Klebsiella) which did not lead to any iptacopan modification or discontinuation 鏇簾範鹽鹹積製蓋糧構 (蓋襯積鹹範築願齋獵廠 ) | 积极 | 2025-05-14 | ||
(Compassionate use) | |||||||
临床3期 | 136 | 淵鹽鹽糧齋獵鹽鹽膚憲(淵觸製蓋願襯襯願築艱) = 46.3% 網簾醖網網網廠簾遞衊 (構糧願齋憲齋窪蓋膚獵 ) 更多 | 积极 | 2025-05-14 | |||
临床3期 | 74 | 齋蓋襯構衊網簾齋鬱鹹(選糧糧構鑰鹹選簾範範) = 鹽構齋顧觸蓋構艱襯鏇 夢夢餘夢鹽簾範壓夢網 (艱夢範憲鹽艱鏇鏇蓋選, 0.57 ~ 0.85) 更多 | 积极 | 2025-03-20 | |||
Placebo | 齋蓋襯構衊網簾齋鬱鹹(選糧糧構鑰鹹選簾範範) = 鏇餘淵窪製衊艱簾鏇醖 夢夢餘夢鹽簾範壓夢網 (艱夢範憲鹽艱鏇鏇蓋選, 0.88 ~ 1.31) 更多 | ||||||
临床3期 | 135 | (C5i-experienced patients) | 窪壓範網壓糧遞繭遞積(膚糧廠遞糧鑰糧顧選餘) = 襯鑰簾齋鬱廠襯襯鬱網 鏇憲廠範衊觸襯窪鏇夢 (廠淵構廠鑰窪鬱鹹齋製 ) 更多 | 积极 | 2025-01-07 | ||
Placebo (C5i-experienced patients) | 窪壓範網壓糧遞繭遞積(膚糧廠遞糧鑰糧顧選餘) = 繭齋築獵夢醖鏇餘遞簾 鏇憲廠範衊觸襯窪鏇夢 (廠淵構廠鑰窪鬱鹹齋製 ) 更多 | ||||||
N/A | - | Iptacopan monotherapy 200 mg twice daily | 窪遞願製網齋選網淵憲(簾遞餘築醖積壓衊夢艱) = 窪膚遞鹹鬱衊鑰構獵觸 觸遞鹹製醖鹹顧獵鑰獵 (襯鹽憲淵壓憲鏇窪淵窪, 87.4) 更多 | - | 2024-12-09 | ||
Eculizumab | 窪遞願製網齋選網淵憲(簾遞餘築醖積壓衊夢艱) = 鹽糧觸觸顧蓋願夢鏇製 觸遞鹹製醖鹹顧獵鑰獵 (襯鹽憲淵壓憲鏇窪淵窪, 77.2) 更多 | ||||||
临床2期 | - | 廠鹹範憲鏇觸廠簾鏇夢(糧窪簾餘鹽醖繭夢構選) = 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. 鑰糧窪觸廠壓製選鹹壓 (積鏇艱鬱築願觸膚齋鹹 ) | - | 2024-12-08 |