About Fabrazyme

Fabrazyme^® is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha-galactosidase deficiency). It is produced by recombinant DNA technology using mammalian Chinese Hamster Ovary (CHO) cell culture, a well-characterised cell line used to produce numerous therapeutic human recombinant DNA proteins. This includes Cerezyme^® (imiglucerase), an enzyme replacement therapy that has safely and effectively treated over 4,000 patients with Gaucher disease, another debilitating lysosomal storage disease. Other CHO derived products are: Neorecormon^®, Refacto^®, Avonex^®, Enbrel^®, etc.

Please see the full prescribing information for Fabrazyme®.

For more information on the criteria for reimbursement of Fabrazyme^® in Belgium, see RIZIV/INAMI reimbursement criteria for Fabrazyme®.

To access information about the steps required to reconstitute and administer Fabrazyme^®, see Reconstitution & Administration Fabrazyme®

Please report all adverse events promptly to Genzyme Belgium by completing an Adverse Event Form and fax it to +32 2 714 17 09. Please Contact Us with any questions.

How does Fabrazyme^® work?

Fabrazyme^® facilitates the breakdown of globotriaosylceramide (GL-3) and other glycosphingolipids to treat the underlying cause of the disease. It is richly sialylated and has appropriate expression of mannose-6-phosphate to ensure lysosomal uptake.

Two placebo-controlled, double-blind trials have been conducted as part of the clinical development program. A first study to evaluate the efficacy on a biomarker (surrogate endpoint) [1], and a second to prove that such efficacy correlates with major clinical endpoints (renal, cardiac, or cerebrovascular events, or death) [2].

These two studies have shown that biweekly infusions of Fabrazyme^® in a dose of 1 mg/kg resulted in near complete clearance of vascular endothelium in the kidney (the primary endpoint in the first study) as well as significantly reduces the time to a first clinical event (the primary endpoint used in the second study)(see table I).

Table I: Placebo-controlled Fabrazyme^® clinical trials

* Fabrazyme 1 mg/kg every other week

References

1. Eng CM, Guffon N, Wilcox WR, Germain DP, Lee P, Waldek S, Caplan L, Linthorst GE, Desnick RJ. Safety and efficacy of recombinant human α-galactosidase A-replacement therapy in Fabry’s disease. N Engl J Med 2001b; 345(1):9-16.

2. Banikazemi M, et al. Agalsidase-Beta Therapy for Advanced Fabry Disease – A Randomized Trial Annals of Internal Medicine. 2007; 146; 77-86