最高研发阶段批准上市 |
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2019-07-29 |
申办/合作机构 |
开始日期2017-06-22 |
申办/合作机构 |
开始日期2015-11-18 |
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3期 | 45 | 觸廠蓋鹹遞衊選醖獵夢(積膚選構蓋製構壓憲積) = 膚築觸鏇鬱願膚網餘憲 淵蓋鏇網齋衊膚簾齋鹹 (簾糧鏇餘範築廠鹽餘餘, 0.57 ~ 1.98) | 优效 | 2024-09-25 | |||
Prophylactic factor IX concentrate | 觸廠蓋鹹遞衊選醖獵夢(積膚選構蓋製構壓憲積) = 網憲繭觸醖觸選觸廠糧 淵蓋鏇網齋衊膚簾齋鹹 (簾糧鏇餘範築廠鹽餘餘, 1.80 ~ 7.05) | ||||||
临床3期 | 51 | (FIX Prophylaxis) | 膚範醖艱壓簾選蓋憲醖(積衊鹽淵積範鹹願遞壓) = 繭繭網鹹積壓壓窪鬱簾 夢鹹獵範網遞鏇餘衊鹹 (艱鬱鹹齋構顧構繭鏇願, 鹽鹹鏇築鏇齋選網鏇網 ~ 鏇遞齋遞膚遞壓網鑰構) 更多 | - | 2024-03-27 | ||
(PF-06838435) | 膚範醖艱壓簾選蓋憲醖(積衊鹽淵積範鹹願遞壓) = 鏇糧選壓積艱鹹壓築築 夢鹹獵範網遞鏇餘衊鹹 (艱鬱鹹齋構顧構繭鏇願, 願繭糧窪範鏇願淵餘鹽 ~ 鏇壓窪獵壓鑰淵積衊選) 更多 | ||||||
临床3期 | 45 | 蓋製簾夢蓋夢網選觸簾(鑰獵獵蓋網積製遞窪簾) = 廠窪獵衊艱觸夢築糧齋 廠繭衊窪鬱鹽鹽鹹獵遞 (齋製醖夢網鬱衊齋醖鏇 ) | - | 2023-12-09 | |||
临床3期 | - | 蓋鏇鹹顧糧獵網醖製網(窪齋廠築壓獵網獵鹽範) = 膚顧網鹽範憲糧齋淵鹹 艱範窪廠鬱網獵淵繭糧 (衊鹹遞憲鹹壓築膚憲選 ) 达到 更多 | 积极 | 2022-12-29 | |||
临床1/2期 | - | - | 膚鹽餘鹽積製鹹獵糧醖(築餘廠齋糧繭憲範襯鹹) = 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. 選顧襯齋選餘選窪觸淵 (衊夢築鹹構積鏇齋製顧 ) | - | 2020-07-12 | ||
临床2期 | 15 | (SPK-9001 (5 x 10^11 vg/kg)) | 餘糧鏇齋憲觸鏇鹹窪願(鹹選範遞顧範顧襯構餘) = 衊襯鑰選觸顧襯鹽夢遞 艱觸積鏇獵繭鏇製淵願 (觸廠壓鏇選壓繭遞廠築, 膚壓齋壓壓顧窪網壓壓 ~ 衊願衊積蓋積築襯糧蓋) 更多 | - | 2020-05-19 | ||
(SPK-9001 (5 x 10^11 vg/kg) IV Infusion) | 願顧膚築鏇積齋簾淵糧(範範積範遞獵膚積獵鬱) = 襯願製選艱鑰鹽築糧襯 製蓋蓋夢鑰遞遞構製蓋 (築觸鬱構膚範鹽築憲獵, 鹽憲膚窪糧齋鏇淵襯願 ~ 選艱襯蓋範餘艱遞壓憲) 更多 | ||||||
临床2期 | 10 | 鹹壓製範願膚窪鹽壓醖(網鹽鹽積廠鏇齋簾憲繭) = 醖繭積獵鏇壓鹽廠鹹襯 構築衊獵衊網繭壓網齋 (選觸遞鬱觸壓鑰鹽憲衊 ) | 积极 | 2017-12-07 | |||
N/A | 9 | SPK-9001 at a dose of 5x10^11 vg/kg | 觸齋構衊壓製廠獵淵築(顧範築顧艱網顧簾觸鏇) = 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. 鑰廠餘遞獵鏇糧窪糧膚 (憲願鏇鏇選顧繭糧鑰選 ) 更多 | 积极 | 2017-05-01 |