Keating Gillian M, Simpson Dene
Wolters Kluwer Health, Adis, Auckland, New Zealand.
Drugs. 2007;67(3):435-55. doi: 10.2165/00003495-200767030-00007.
Agalsidase beta (Fabrazyme) is a recombinant human alpha-galactosidase A enzyme approved for intravenous use in the treatment of Fabry disease. Fabry disease is a progressive, multisystemic, potentially life threatening disorder caused by a deficiency of alpha-galactosidase A. This deficiency results in accumulation of glycosphingolipids, particularly globotriaosylceramide (GL-3), in the lysosomes of various tissues. This accumulation is the underlying driver of disease progression. Agalsidase beta provides an exogenous source of alpha-galactosidase A.Intravenous agalsidase beta is effective and well tolerated in patients with Fabry disease. In a phase III trial, agalsidase beta was shown to clear GL-3 from various target cells and, in a subsequent extension of this trial, prevent GL-3 reaccumulation. In a post-approval trial, agalsidase beta was shown to provide significant clinical benefit by reducing the risk of a major clinical event. Thus, agalsidase beta represents an important advance in the treatment of Fabry disease, and agalsidase beta therapy should be strongly considered in patients with Fabry disease who are suitable candidates.
阿加糖酶β（法布赞）是一种重组人α-半乳糖苷酶A，已被批准静脉注射用于治疗法布里病。法布里病是一种进行性、多系统、可能危及生命的疾病，由α-半乳糖苷酶A缺乏引起。这种缺乏导致糖鞘脂，特别是球三糖神经酰胺（GL-3）在各种组织的溶酶体中积累。这种积累是疾病进展的根本驱动因素。阿加糖酶β提供了α-半乳糖苷酶A的外源性来源。静脉注射阿加糖酶β对法布里病患者有效且耐受性良好。在一项III期试验中，阿加糖酶β被证明可从各种靶细胞中清除GL-3，并且在该试验的后续扩展中，可防止GL-3重新积累。在一项批准后试验中，阿加糖酶β被证明通过降低重大临床事件的风险提供显著的临床益处。因此，阿加糖酶β代表了法布里病治疗的一项重要进展，对于适合的法布里病患者应强烈考虑阿加糖酶β治疗。
