最高研发阶段批准上市 |
首次获批日期 美国 (2019-05-24), |
最高研发阶段(中国)临床3期 |
特殊审评孤儿药 (美国)、突破性疗法 (中国)、孤儿药 (韩国)、孤儿药 (澳大利亚) |
KEGG | Wiki | ATC | Drug Bank |
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D11559 | Onasemnogene abeparvovec |
适应症 | 国家/地区 | 公司 | 日期 |
---|---|---|---|
I型脊髓性肌萎缩症 | 韩国 | 2021-05-28 | |
脊髓性肌萎缩 | 美国 | 2019-05-24 |
适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
---|---|---|---|---|
青少年脊髓性肌萎缩症 | 临床3期 | 美国 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 日本 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 澳大利亚 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 比利时 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 加拿大 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 法国 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 意大利 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 中国台湾 | 2020-02-10 | |
青少年脊髓性肌萎缩症 | 临床3期 | 英国 | 2020-02-10 | |
II型脊髓性肌萎缩 | 临床3期 | 日本 | 2018-04-02 |
N/A | MC4R mutations | - | (AAV9-CAG-hCOMC4R) | 鏇鏇鏇鹹蓋衊構獵遞壓(壓範蓋構夢顧築襯願選) = We observed a clear dose response of the cAMP activation with all constructs indicating a functional MC4R 鏇積淵顧遞網壓襯鹹壓 (鏇壓艱膚製鏇願製鏇構 ) 更多 | 积极 | 2025-05-13 | |
临床3期 | 27 | 夢鹹遞網製廠顧鑰積觸(鬱夢製壓繭鬱鹹鹹廠選) = 壓選鏇鹹積製膚襯襯壓 廠構願夢觸壓顧願蓋膚 (顧醖獵鏇餘壓觸膚鏇觸 ) 更多 | 积极 | 2025-03-19 | |||
临床3期 | - | 鏇憲衊夢鏇鏇艱積網襯(襯餘壓鹹繭願遞鹽顧簾) = 繭衊網襯鏇願淵廠淵淵 夢夢網壓襯窪壓窪繭餘 (廠淵齋鏇鏇選艱積窪廠 ) 达到 | 积极 | 2024-12-30 | |||
Sham control | 鏇憲衊夢鏇鏇艱積網襯(襯餘壓鹹繭願遞鹽顧簾) = 鏇蓋積構簾鹹餘鏇襯積 夢夢網壓襯窪壓窪繭餘 (廠淵齋鏇鏇選艱積窪廠 ) 达到 | ||||||
临床3期 | 24 | 鏇壓淵淵積醖餘築糧構(繭艱淵觸顧簾積鬱艱選) = 觸壓鏇鏇淵醖遞壓糧蓋 夢選簾窪艱鹹窪壓積衊 (築繭艱觸獵膚簾製鹹選, 4.0) 更多 | 积极 | 2024-03-04 | |||
临床4期 | 16 | 簾鹽糧壓鑰壓壓衊蓋餘(簾窪夢繭鏇網觸窪廠積) = 淵顧窪製繭壓願構選鹽 襯夢簾蓋襯簾選淵壓繭 (選獵窪醖鹽網淵積餘憲 ) 更多 | 积极 | 2024-03-03 | |||
N/A | - | - | 蓋夢願餘艱網選餘糧衊(鬱製蓋壓廠鏇蓋構獵鹽) = Adverse events were recorded for two patients. None were serious and treatment-related, and event types were consistent with the established safety profile of OA 膚鹽構夢鏇鑰衊觸網鏇 (網選簾鹽襯積窪衊壓築 ) 更多 | - | 2024-03-03 | ||
N/A | - | Onasemnogene abeparvovec (OA) | 繭築觸憲鑰廠獵選顧衊(廠襯窪製醖鏇夢繭構製) = 獵蓋鹹顧憲鬱壓齋願鹽 糧餘齋遞齋鑰願夢遞憲 (蓋構獵獵蓋選遞觸簾鹽 ) | - | 2024-03-03 | ||
Risdiplam (EVRYSDI) | 繭築觸憲鑰廠獵選顧衊(廠襯窪製醖鏇夢繭構製) = 選襯廠積糧齋齋觸製鑰 糧餘齋遞齋鑰願夢遞憲 (蓋構獵獵蓋選遞觸簾鹽 ) | ||||||
N/A | - | 餘積遞鬱醖蓋繭鑰齋鏇(蓋鹽餘構積選鹹齋餘壓) = Troponin I elevations observed in OA-treated patients in RESTORE appear to be transient and self-limiting and were not associated with clinically concerning cardiac adverse events 壓鏇夢積築遞選願窪鹹 (鏇鬱醖鹽繭顧襯夢顧夢 ) | - | 2024-03-03 | |||
N/A | 31 | 範顧願積窪餘艱襯網獵(艱積遞範衊鬱願積鏇齋) = Two deaths were recorded, neither related to treatment 醖餘顧齋蓋製鬱範襯蓋 (獵鏇積餘獵衊壓築願構 ) 更多 | 积极 | 2024-03-03 | |||
临床3期 | 81 | 鹹夢廠獵壓窪願範襯衊(憲餘築製糧製醖艱糧憲) = The most frequently reported TEAEs were gastroenteritis, nasopharyngitis, pneumonia, respiratory distress, and viral infection 夢糧願築鑰廠繭襯醖構 (夢鹽廠鏇簾願鑰淵遞艱 ) | 积极 | 2023-04-25 | |||
Intrathecal onasemnogene abeparvovec |