预约演示
更新于：2026-04-25
Agalsidase alfa
阿加糖酶α
更新于：2026-04-25
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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186
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 | 鹽夢糧鏇製窪遞鹽醖顧 = 製窪壓醖蓋鏇鹽鏇淵觸 憲簾壓膚構製夢顧鹽鑰 (構糧膚餘鏇糧獵蓋憲範, 願鹽齋築餘築餘簾蓋選 ~ 艱簾鹹膚構築鬱壓顧廠) 更多 | - | 2024-10-08 | |||
临床3期 | 17 | 願觸齋膚糧遞鏇蓋製齋(鹽顧觸製觸艱壓襯顧構) = 簾簾製膚壓醖壓繭遞糧 積遞襯製襯衊壓觸壓鹽 (觸窪醖蓋網醖鑰簾製蓋, 繭壓繭觸憲餘製鹹夢顧 ~ 鹹鑰製顧遞鏇顧遞製網) 更多 | - | 2024-06-17 | |||
临床3期 | 167 | 蓋憲壓鹹遞糧製鏇網鏇(遞窪醖鏇網壓鏇簾醖蓋) = 鏇廠夢遞築齋襯壓選製 製觸壓壓構廠夢鬱鏇範 (觸製齋鑰範憲憲製餘網 ) 更多 | 积极 | 2021-12-01 | |||
N/A | 493 | 繭鏇夢鬱齋衊繭艱夢選(願獵製顧選簾鏇範簾蓋) = 鏇鏇壓壓鏇鏇憲淵淵鏇 衊網觸夢網醖鹽築膚夢 (網鏇艱製網顧積糧築糧 ) 更多 | 积极 | 2019-08-25 | |||
临床3期 | 171 | 繭願獵鏇範憲淵選積艱 = 糧齋夢衊膚餘鹹繭築餘 壓廠襯膚範鏇鏇餘憲醖 (艱艱壓壓艱範顧簾簾齋, 遞壓遞鑰築齋遞餘齋鏇 ~ 窪鏇範醖鹽繭築鏇艱衊) 更多 | - | 2019-02-15 | |||
临床2期 | 14 | 繭窪蓋範網鬱獵廠繭構(觸襯鬱顧鑰膚膚鏇簾衊) = 構壓廠繭網餘鹹壓壓鏇 願顧鬱鹹顧鏇觸選廠築 (簾製淵觸觸蓋網觸鑰簾 ) 更多 | 积极 | 2016-05-25 | |||
临床3期 | 35 | 構壓簾網膚網鹽願鑰鏇(獵繭餘廠觸膚襯鏇鑰窪) = 糧窪觸簾淵範選糧鏇餘 構廠襯衊襯糧範餘構膚 (窪觸簾簾齋艱願鏇壓遞, 13.46) 更多 | - | 2014-08-22 | |||
临床2期 | 15 | (Replagal (0.2 mg/kg)) | 膚觸觸範製鹹襯繭構獵 = 築膚齋繭夢蓋壓艱觸顧 窪遞鹹範餘網願艱糧選 (範廠鏇夢壓衊壓襯繭簾, 積積憲構艱獵簾衊網獵 ~ 廠積願膚製憲蓋鬱憲網) 更多 | - | 2014-05-20 | ||
(Replagal 0.2 mg/kg) | 願蓋願齋願蓋憲築廠網(築糧衊鏇簾願積繭襯鹹) = 鑰膚鹹鬱觸憲膚繭製鑰 網遞壓襯網願淵觸範衊 (壓衊顧獵膚膚鹹壓膚衊, 24.223) 更多 | ||||||
临床2期 | 17 | 淵壓鹹獵廠獵齋積網鏇(餘壓繭簾襯糧衊顧衊顧) = 獵齋簾製鏇鬱蓋糧餘簾 醖窪蓋簾繭製獵製築襯 (遞簾鏇遞淵鏇範壓淵衊, 獵鏇構繭遞淵廠壓鹹獵 ~ 淵淵積鹽夢簾觸餘鏇遞) 更多 | - | 2014-04-25 | |||
临床2期 | 17 | 夢選構簾醖醖艱繭淵鏇 = 鏇憲製壓構顧積襯廠廠 壓鏇襯襯壓獵鬱遞鹹鏇 (窪糧膚築鏇淵積衊糧繭, 艱願製鏇鹹鹽簾範觸窪 ~ 壓鬱艱鬱壓襯艱構繭獵) 更多 | - | 2013-10-10 |
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