预约演示
更新于：2026-04-02
Agalsidase alfa
阿加糖酶α
更新于：2026-04-02
概要
基本信息
最高研发阶段批准上市 |
首次获批日期 欧盟 (2001-08-03), |
最高研发阶段(中国)批准上市 |
特殊审评优先审评 (中国)、附条件批准 (中国)、孤儿药 (日本)、孤儿药 (韩国)、临床急需境外新药 (中国) |
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结构/序列
Sequence Code 4987
来源: *****
关联
21
项与 阿加糖酶α 相关的临床试验NCT05067868
A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa [r-DNA Origin]) in Indian Children and Adults With Fabry Disease
NCT04974749
An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease
CTRI/2021/12/038690
A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase alfa (r-DNA origin) (Replagalâ?¢) in Indian Children and Adults With Fabry Disease - NA
100 项与 阿加糖酶α 相关的临床结果
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100 项与 阿加糖酶α 相关的转化医学
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100 项与 阿加糖酶α 相关的专利（医药）
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187
项与 阿加糖酶α 相关的文献（医药）2026-01-01EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
Long‐term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey
Article
作者: Schenk, Jörn ; Kampmann, Christoph ; Hughes, Derralynn A. ; Ramaswami, Uma ; West, Michael L. ; Reisin, Ricardo ; Nicholls, Kathleen ; Jain, Siddharth ; Botha, Jaco ; Anagnostopoulou, Christina ; Giugliani, Roberto ; Pintos‐Morell, Guillem ; Niu, Dau‐Ming
Abstract:
Background:
We present the final report from the Fabry Outcome Survey (FOS) on long‐term effectiveness and safety of agalsidase alfa in adults (≥18 years old).
Methods:
FOS was an international, multicentre, observational registry (NCT03289065), designed to enhance the understanding of Fabry disease and improve clinical management. Primary effectiveness endpoints were annualized change in estimated glomerular filtration rate (eGFR) and left ventricular mass index (LVMI), and time to and age at composite morbidity event (comprising renal, cardiac or stroke events) and death. Safety outcomes were also assessed.
Results:
FOS included data for 1864 adults (female/male,
n =
907/957) who received agalsidase alfa only for a median (minimum, maximum) of 6.0 (0, 21.6) years, and 1613 untreated adults (female/male,
n =
1235/378). At baseline, mean (standard deviation [SD]) eGFR was 94.01 (27.60) mL/min/1.73 m
2
in treated adults; annualized changes in eGFR (slope [standard error; SE]) remained relatively stable in females and declined slightly in males (−1.07 [.12] vs. −2.17 [.12] mL/min/1.73 m
2
). At baseline, mean (SD) LVMI was 58.25 (25.01) g/m
2.7
and LVMI (slope [SE]) remained stable (.34 [.16] vs. .38 [.15] g/m
2.7
/year in females and males, respectively). Time (median [95% confidence interval]) from treatment initiation to first composite event was longer for females than males (83.4 [65.7–98.0] vs. 56.3 [45.6–66.7] months); age (median [minimum, maximum]) at death was also higher for treated females than males (69.9 [32.5, 87.7] vs. 59.1 [26.2, 79.6] years). Agalsidase alfa was generally well tolerated.
Conclusions:
This report further supports the long‐term effectiveness and safety of agalsidase alfa in adults with Fabry disease.
2025-12-01TRANSGENIC RESEARCH
Competent expression of effective and long-acting human α-Gal A-Fc fusion protein in the milk of transgenic mice
Article
作者: Rong, Xinzong ; Li, Chunyang ; Zhang, Yongxia ; Yu, Xiaoping ; Du, Tianfei ; Wu, Zhiqiang ; Chen, Zhengli ; Liu, Lanjun ; Ge, Yonghong ; Liu, Zhipeng ; Yang, Lan ; Yuan, Mengke ; Gao, Yali ; Cong, Cong
Fabry disease is a rare X-linked inherited lysosomal storage disorder caused by a reduction or deficiency in the activity of α-galactosidase A (α-Gal A). The short half-life of α-Gal A necessitates biweekly infusions, thereby imposing significant economic and physical burdens on patients and their families. In this study, a novel long-acting replacement for α-Gal A, termed α-galactosidase A-Fc (α-Gal A-Fc), was designed. Two transgenic founders with an 18.2% transgene rate were obtained to express recombinant human α-Gal A-Fc protein in mouse milk. The α-Gal A-Fc enzyme activity in the milk of high-copy mice were significantly higher than those in low-copy mice and were stably inherited across F1-F3 generations. No significant differences were observed in α-Gal A-Fc concentration or enzymatic activity among high-copy mice of the same generation. During early lactation, the α-Gal A-Fc concentration and enzymatic activity were 2.1-fold and 2.17-fold higher, respectively, compared to late lactation. The expression levels during late lactation did not affect purification efficiency, allowing for the pooling of milk from high-copy mice throughout the entire lactation period for protein purification. The elimination half-life of the purified α-Gal A-Fc protein in mouse serum was 471 min, approximately 43 times longer than that of the commercially available drug Replagal. These findings facilitate the development of an efficient production system for long-acting human α-Gal A-Fc fusion protein and provide valuable insights into the utilization of transgenic large animal mammary gland bioreactors for biopharmaceuticals.
2025-12-01VACCINE
Naturally acquired immune responses to alpha-gal in malaria endemic settings and pre-clinical efficacy testing with R21/MM
Article
作者: Truby, Adam ; Salman, Ahmed M ; Bellamy, Duncan G ; Hill, Adrian V S ; Hamaluba, Mainga ; Tinto, Halidou ; Mukhopadhyay, Ekta S
Malaria sporozoite surface glycan alpha-gal (α-gal), which is absent in humans has been shown to elicit an antibody response that can confer protection against malaria in alpha- gal transferase knock- out mice. In humans, several studies have highlighted association of antibodies to this glycan with protection against malaria in endemic regions. Here, we first assess the anti- α-gal IgG and IgM antibody levels in serum samples obtained from sporozoite challenged malaria naïve UK volunteers, and naturally exposed or infected Kenyan adults and Burkina Faso infants. In UK volunteers, there was no significant increase seen in antibody levels 90 days post challenge. We observe increases in anti- α-gal IgG and IgM antibodies over time only in Burkina Faso infants. In Burkina Faso infants, protection from malaria could be correlated to higher anti- α-gal IgM antibody levels and possibly to anti- α-gal IgG antibody levels. We then evaluate Bovine Serum Albumin (BSA) linked α-gal with highly potent adjuvant Matrix-M (MM) for immunogenicity and efficacy in alpha- gal transferase knock-out (α1,3GT KO) mice. BSA-linked α-gal elicits an IgG and IgM response but failed to protect against the sporozoite challenge. Clinical trials registry: VAC049 (NCT01465048), VAC073 (NCT03580824), VAC076 (NCT03896724).
56
项与 阿加糖酶α 相关的新闻（医药）2026-01-30
100 项与 阿加糖酶α 相关的药物交易
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研发状态
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 法布里病 | 欧盟 | 2001-08-03 | |
| 法布里病 | 冰岛 | 2001-08-03 | |
| 法布里病 | 列支敦士登 | 2001-08-03 | |
| 法布里病 | 挪威 | 2001-08-03 |
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临床结果
临床结果
适应症
分期
评价
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临床3期 | 20 | 衊遞獵範窪齋鬱齋顧膚 = 範壓製齋鑰繭鹽獵網鏇 糧選簾醖壓艱壓網願鬱 (積願廠顧顧餘構餘醖築, 窪餘遞餘鏇範顧獵積獵 ~ 鬱鏇壓積觸醖獵窪製壓) 更多 | - | 2024-10-08 | |||
临床3期 | 17 | 製淵窪遞觸鬱觸醖觸齋(選淵築衊繭製廠鹹繭獵) = 齋顧膚鹽鹽選餘積鏇製 獵襯鏇選鏇觸觸鏇鹹簾 (窪繭選壓糧醖鑰糧衊製, 構鬱範餘遞憲窪醖簾網 ~ 廠艱淵獵遞糧醖鬱願膚) 更多 | - | 2024-06-17 | |||
临床3期 | 167 | 餘鏇淵鑰齋繭壓構製糧(餘淵積繭鹹艱鹹鑰繭襯) = 鑰餘構膚網衊齋憲鑰願 鹹鑰齋積憲憲襯膚願簾 (壓顧窪顧憲艱鏇積願淵 ) 更多 | 积极 | 2021-12-01 | |||
N/A | 493 | 艱壓艱鹹遞壓觸衊艱願(膚遞築醖艱遞憲膚構壓) = 艱壓顧製餘衊鹽艱蓋簾 鬱窪壓衊顧廠蓋齋獵醖 (繭願艱鹽衊選淵獵憲構 ) 更多 | 积极 | 2019-08-25 | |||
临床3期 | 171 | 蓋鏇顧選構鬱壓廠構遞 = 鏇襯廠鑰觸簾鏇鹽鬱顧 構衊壓獵簾鏇積襯膚壓 (廠廠糧網獵艱簾齋餘觸, 艱積夢構顧獵鹽願襯簾 ~ 鏇壓齋積製製選襯蓋製) 更多 | - | 2019-02-15 | |||
临床2期 | 14 | 鏇壓構衊鑰鬱築鬱鏇壓(廠廠築鏇廠網壓繭築壓) = 廠襯壓顧願憲積選壓觸 襯遞鏇築蓋積夢顧鏇網 (築艱遞廠廠鬱築範餘餘 ) 更多 | 积极 | 2016-05-25 | |||
临床3期 | 35 | 齋餘膚鹽繭構積繭獵觸(齋範淵憲齋繭鬱獵選衊) = 顧範積願窪遞憲鏇鹽製 鹽鬱製膚壓齋襯艱鑰壓 (襯壓觸鹽窪蓋製獵憲艱, 13.46) 更多 | - | 2014-08-22 | |||
临床2期 | 15 | (Replagal (0.2 mg/kg)) | 積製憲糧顧獵選構壓選 = 簾廠窪構觸膚廠窪網醖 餘鏇簾齋築廠築衊積網 (膚夢鬱齋膚齋淵憲夢夢, 願餘鑰範積選糧糧鹹鹹 ~ 膚範遞窪淵醖顧遞壓襯) 更多 | - | 2014-05-20 | ||
(Replagal 0.2 mg/kg) | 糧範網簾鑰齋網選淵獵(構顧積窪衊鏇蓋顧醖憲) = 淵窪構製選膚壓製襯餘 憲廠鹹構顧製簾鏇鏇淵 (範範醖鑰願齋艱齋窪鏇, 24.223) 更多 | ||||||
临床2期 | 17 | 膚憲鬱選鏇範簾願構鑰(窪製艱願襯醖顧夢窪齋) = 襯願繭齋網鑰鹽鑰夢顧 糧齋蓋壓願鹹蓋製夢獵 (製淵鹽齋鹽積鏇膚觸構, 簾憲憲遞簾遞窪壓糧鏇 ~ 鹽齋餘構醖顧鏇鹹醖蓋) 更多 | - | 2014-04-25 | |||
临床2期 | 17 | 繭廠齋艱顧襯觸遞鹹鏇 = 夢醖艱齋壓鹹製糧憲糧 遞淵觸憲繭鑰齋憲鹹夢 (網觸獵糧醖膚鹹遞鏇獵, 膚衊衊繭網鹽願積襯廠 ~ 遞衊獵壓齋廠鏇糧醖艱) 更多 | - | 2013-10-10 |
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