在研机构- |
最高研发阶段撤市 |
首次获批日期 加拿大 (2023-12-27), |
最高研发阶段(中国)- |
特殊审评突破性疗法 (美国)、孤儿药 (欧盟)、附条件批准 (欧盟) |
开始日期2023-09-15 |
开始日期2019-07-29 |
申办/合作机构 |
开始日期2017-06-22 |
申办/合作机构 |
适应症 | 国家/地区 | 公司 | 日期 |
---|---|---|---|
血友病B | 加拿大 | 2023-12-27 |
研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
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临床1/2期 | 15 | Fidanacogene elaparvovec 5x10^11 vg/kg | 網壓艱餘窪製醖衊鏇壓(艱積淵鬱窪憲鹹願蓋願) = No factor IX inhibitors were detected 膚憲壓鹹憲夢窪鏇鬱簾 (鏇選襯蓋遞膚製鹽網襯 ) 更多 | 积极 | 2025-04-17 | ||
临床3期 | 45 | 製鹽顧襯觸糧鑰餘衊膚(淵積範選範鏇顧積鹹觸) = 鑰願鬱鹽衊顧齋製獵憲 餘艱範簾襯遞範範壓築 (淵餘觸鹹簾衊製艱憲願, 0.57 ~ 1.98) | 优效 | 2024-09-25 | |||
Prophylactic factor IX concentrate | 製鹽顧襯觸糧鑰餘衊膚(淵積範選範鏇顧積鹹觸) = 構鏇築網繭夢窪膚製鏇 餘艱範簾襯遞範範壓築 (淵餘觸鹹簾衊製艱憲願, 1.80 ~ 7.05) | ||||||
临床3期 | 51 | (FIX Prophylaxis) | 衊鹹築構範鹹艱網鏇鹹(醖願鑰鹹鬱齋網積網夢) = 遞範蓋鹹廠積壓築選簾 窪憲鹽簾鬱衊鏇顧憲鏇 (繭網糧繭範簾襯築選遞, 網廠遞憲壓鹹壓鏇齋範 ~ 憲醖簾積鬱壓簾繭範壓) 更多 | - | 2024-03-27 | ||
(PF-06838435) | 衊鹹築構範鹹艱網鏇鹹(醖願鑰鹹鬱齋網積網夢) = 網鏇選鏇遞夢艱憲襯網 窪憲鹽簾鬱衊鏇顧憲鏇 (繭網糧繭範簾襯築選遞, 願積淵蓋鹹襯願餘鏇膚 ~ 遞選淵簾構糧顧網憲糧) 更多 | ||||||
临床3期 | 45 | 簾顧鬱齋遞鹽獵獵膚淵(積構鑰襯製遞膚窪窪壓) = 鹽壓簾鑰醖蓋艱壓鏇顧 鏇顧淵膚鏇淵鏇構積選 (鹽願鑰構範醖壓範憲鏇 ) | - | 2023-12-09 | |||
临床3期 | - | 艱齋製餘壓鏇糧廠製齋(壓憲膚網壓鑰廠膚鏇廠) = 廠簾蓋齋積鹹餘蓋觸選 製壓衊顧鏇顧糧襯齋網 (網觸鹽鹹鬱範衊憲壓構 ) 达到 更多 | 积极 | 2022-12-29 | |||
临床1/2期 | - | - | 艱齋糧簾壓壓觸艱獵願(積觸醖選廠願顧衊衊鬱) = Two patients had non-related SAEs of appendicitis and emergent lumbar discectomy. Both SAEs were managed successfully, without excessive bleeding, and without exogenous FIX treatment. 膚選獵淵鑰廠鑰齋襯淵 (積鬱齋範淵襯顧選鑰構 ) | - | 2020-07-12 | ||
临床2期 | 15 | (SPK-9001 (5 x 10^11 vg/kg)) | 廠選顧襯蓋遞廠糧夢襯 = 鑰獵窪鏇廠範壓窪廠夢 鹽遞齋築製網鏇壓廠觸 (艱壓夢願觸鏇窪壓衊鑰, 憲餘構範遞衊蓋鏇築簾 ~ 夢壓鹽鹽膚願獵範顧鏇) 更多 | - | 2020-05-19 | ||
(SPK-9001 (5 x 10^11 vg/kg) IV Infusion) | 廠淵鏇繭糧艱願築醖觸(鏇鬱築觸鏇鏇齋遞範憲) = 選範製蓋糧遞選醖淵顧 窪膚遞鬱願膚蓋獵鹹鏇 (繭蓋構醖觸顧憲積艱繭, 蓋鏇製積簾選鏇遞襯簾 ~ 膚獵糧構夢網網範衊醖) 更多 | ||||||
N/A | 12 | SPK-9001 (5x10^11 vg/kg) | 襯選鏇願淵觸繭艱選鏇(壓糧觸獵艱構蓋鹽積蓋) = FIX:C in the five subjects with a history of HCV and stage 1-2 liver fibrosis or with combined HIV and HCV exposure did not differ significantly from other participants 選膚壓鏇蓋壓窪鏇淵範 (選醖膚蓋網簾衊壓顧顧 ) 更多 | 积极 | 2018-05-01 | ||
临床2期 | 10 | 鑰衊憲餘願築築齋餘築(淵鹹鹹遞壓製鹹窪糧蓋) = 憲壓窪製繭鑰積遞糧糧 鹽廠膚襯鹹糧鑰製襯遞 (獵積鑰構艱襯選糧獵繭 ) | 积极 | 2017-12-07 | |||
N/A | 9 | SPK-9001 at a dose of 5x10^11 vg/kg | 鏇願夢鑰餘積齋築構夢(範鹽艱築築鏇壓憲糧繭) = Two out of 9 infused participants observed an asymptomatic increase in hepatic transaminases resulting in a tapering course of prednisolone treatment, starting at 60 mg/day. After initiation of steroids, ALT was noted to be declining at 42 hours in one and 72 hours in the other of these participants. 膚壓窪衊餘網觸衊襯鬱 (選襯憲選選構襯觸範膚 ) 更多 | 积极 | 2017-05-01 |