Treatment of Fabry patients > 18 years with enzyme supplementation therapy: comparison of efficacy and toxicity of low dose (0,2 mg/kg) fabrazyme (agalsidase beta) or replagal (agalsidase alfa).

Fabry disease is an X-linked disorder caused by the deficiency of the lysosomal enzyme alfa-Galactosidase A (alfa-Gal). Patients with this disorder suffer in childhood from severe pains in hands and feet and develop severe complications later in life such as renal failure, CVA¡¦s and cardiac complications.

Patients with Fabry disease have a reduced life expectancy. Recently two differently alfa-Galactosidase enzyme preparations have received marketing authorization in the EU (orphan drug).

Conclusions on the differences between these products with regard to safety and efficacy cannot be drawn because of the different dosage and evaluation methods performed.

Therefore treatment should be performed according to a standardized treatment approach which allows comparison of both drugs.

Protocol objectives:

To monitor and evaluate the efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase alpha (FabrazymeTM) and agalsidase beta (ReplagalTM) in an equal dose of 0,2 mg/kg in adults with Fabry disease.

Investigational plan: Symptomatic Fabry patients (aged 18 or older) who fulfil the criteria will receive enzyme therapy for at least 12 months and will be evaluated every 3 months.

Evaluation of efficacy and safety of two different formulas of alfa-Galactosidase A, agalsidase beta (FabrazymeTM) and agalsidase alpha (ReplagalTM) in an equal dose of 0,2 mg/kg in order to detect any differences between these two drugs.

N/A

Patients will receive 0,2mg/kg Fabrazyme (agalsidase beta) or 0,2 mg/kg Replagal (agalsidase alpha), every two weeks for a minumum of 12 months. If there's treatment failure (progression of renal disease, cardiac disease and/or a new cerebral stroke or TIA) during or after this period, patients will be advised to switch to Fabrazyme 1,0 mg/kg/2 wks.