部份中文REPLAGAL处方资料（仅供参考）
Fabry病(法布里病),又称弥漫性体血管角质瘤或糖鞘脂类沉积症。为性连隐性遗传先天性糖鞘磷脂代谢异常病。为α-半乳糖苷酶A缺乏引起糖鞘脂代谢障碍，致使酰基鞘氨酸己之糖苷在组织中积聚而发病。多数于10岁前起病。有四肢疼痛、感觉异常和少汗等表现。皮肤最初为毛细血管扩张，随年龄增长而增多，扩大、呈单个或节状红黑色皮损，压之不褪色，较大皮疹可有过度角化。
Replagal的临床特性：
治疗适应症：
用于治疗α-galactosidase A缺乏患者(即Fabry Disease)，提供长期酵素补充治疗。
使用剂量与方法：
Replagal一次剂量为每公斤体重0.2 mg，以静脉输注至少40分钟，隔周注射一次。对于儿童与青春期患者(0-17岁)或超过65岁患者，还未有剂量调整研究，这些患者使用之安全性与有效性也还未被建立。肝功能受损患者使用之研究还未被建立。肾功能受损患者使用，无须调整剂量。
禁忌症：
对主成份或任何赋型剂产生危急生命之过敏反应者，不可使用。
特殊警语与注意事项：
在一小时内之输注过程中，约有10%患者对Replagal产生轻微、急性之特异反应，一般症状是寒颤及脸部潮红。初次以Replagal治疗患者，以上症状一般约在2-4个月内发生。假如轻微或中度急性输注反应发生，医疗单位应迅速发现并建立适度处理原则。注射应暂时中断5-10分钟，直到症状消退，再继续注射。
轻微及短暂不良反应不须药物治疗或中断注射。除此之外，治疗前一般给予口服antihistamines及(或)corticosteroids，最好注射前1-3小时给药，以避免某些个案随后之反应，症状治疗也是须要的。
任何静脉注射蛋白质产品，过敏性反应都可能发生。假如严重过敏性反应发生，应迅速停止注射Replagal，并应考虑给予适当的症状治疗，也应注意现行紧急治疗之医疗标准。
所有蛋白质药品制剂，蛋白质会被发展为蛋白质抗体，经九个月以Replagal治疗之女性患者，观察到都没有抗体产生之证据，约有55%Replagal治疗之男性患者，观察到会有低的抗体反应，随着约三个月治疗，就会有抗体反应出现。经12-18个月治疗，60%患者自由抗体多于80%产生抗体患者，这足以证明，超过时间抗体还原基础下有免疫耐受性产生。
药物间相互反应：
Replagal不可与chloroquine, amiodarone, benoquin or gentamicin等药物一起使用，它们会抑制细胞间α-galactosidase的活性。α-galactosidase A是一种酵素，它不像细胞色素P450参与药物间反应之后补者。临床研究上，对大部份患者同时使用神经疼痛药物如：carbamazepine, phenytoin,及gabapentin，不会有药物间反应发生。
孕妇及哺乳期妇女用药：
对Replagal而言，无怀孕患者使用之临床报告，对怀孕或器官正要成型之动物作研究，也无直接或间接伤害。也未知Replagal是否会被排泄至人体乳汁中，因此怀孕或授乳妇女使用Replagal应格外小心。

The enzyme replacement therapy agalsidase alfa (Replagal®) has an amino acid sequence identical to that of native α-galactosidase A; intravenous agalsidase alfa 0.2 mg/kg every other week is indicated for the long-term treatment of patients with confirmed Fabry disease. This article reviews the efficacy and tolerability of agalsidase alfa in patients with Fabry disease, as well as summarizing its pharmacologic properties.
Agalsidase alfa had beneficial effects in adult men with Fabry disease, according to the results of two randomized, double-blind, placebo-controlled, 6-month trials (n= 15 and 26). For example, left ventricular mass index was reduced to a significantly greater extent with agalsidase alfa than with placebo. Although the change in myocardial globotriaosylceramide content (primary endpoint in one study) did not significantly differ between agalsidase alfa and placebo recipients, the change in the Brief Pain Inventory (BPI) ‘pain at its worst’ score (reflecting neuropathic pain while without pain medications; primary endpoint in the second study) was improved to a significantly greater extent with agalsidase alfa than with placebo. In addition, the change in creatinine clearance, but not inulin clearance, significantly favored agalsidase alfa versus placebo recipients. Abnormalities in functional cerebral blood flow and cerebrovascular responses were also reversed with agalsidase alfa therapy.
In extensions of these placebo-controlled trials, the reduction in left ventricular mass and improvements in BPI pain scores were maintained after longer-term agalsidase alfa therapy. The significant decline in estimated glomerular filtration rate (eGFR) seen after 48 months’ agalsidase alfa treatment was mainly driven by a marked decline in eGFR seen in four patients with stage 3 chronic kidney disease at baseline (although the progression of decline appeared slower than that seen in historic controls); renal function appeared stable in patients with stage 1 or 2 chronic kidney disease. Certain benefits of agalsidase alfa became apparent with longer-term therapy. For example, a significant reduction in cold and warm detection thresholds and a significant improvement in sweat function were seen after 3 years’ therapy.
Final results from a head-to-head trial comparing the effects of agalsidase alfa and agalsidase beta at approved dosages are not yet available. The only available fully published study compared agalsidase alfa 0.2 mg/kg every other week with an off-label dosage of agalsidase beta 0.2 mg/kg every other week. This randomized, open-label, 24-month trial in adult men and women with Fabry disease generally found no significant differences in outcome between treatment arms. It should be noted that concerns were subsequently raised by the European Medicines Agency regarding the use of agalsidase beta at dosages other than the approved dosage of 1 mg/kg every other week. Preliminary results from an ongoing, randomized, open-label study suggest no differences in outcome between patients with Fabry disease receiving intravenous agalsidase alfa 0.2 mg/kg every other week and those receiving the approved regimen of agalsidase beta 1 mg/kg every other week. In three switching studies, no safety concerns were raised and disease stability was generally maintained following the switch from agalsidase beta 1 mg/kg every other week to agalsidase alfa 0.2 mg/kg every other week.
Agalsidase alfa also demonstrated beneficial effects, including in women and pediatric patients, in non-comparative studies and in the Fabry Outcome Survey.
Agalsidase alfa was generally well tolerated in patients with Fabry disease, with infusion reactions (e.g. rigors, pyrexia, flushing) being the most commonly occurring adverse event. IgG antibodies developed in ≈24% of male patients with Fabry disease who received agalsidase alfa. After 12–54 months of treatment, 17% of agalsidase alfa recipients were still IgG antibody positive, with immunologic tolerance developing in 7% of agalsidase alfa recipients. No IgE antibodies have been detected in any patient receiving agalsidase alfa. No antibody formation was reported in women receiving agalsidase alfa in noncomparative studies.