Fabrazyme
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Active Ingredient: agalsidase beta
Product Overview
About Fabrazyme
Fabrazyme is a solution for infusion (drip into a vein) containing the active substance agalsidase beta.
What it is used for
Fabrazyme is used to treat patients who have Fabry disease, a rare inherited disorder. Patients with Fabry disease do not have enough of an enzyme called alpha-galactosidase A. This enzyme normally breaks down a fatty substance called globotriaosylceramide (GL-3). If the enzyme is not present, GL-3 cannot be broken down and it builds up in the body’s cells, such as kidney cells. People with Fabry disease may have a wide range of signs and symptoms, including severe conditions such as kidney failure, heart problems and stroke.
How it is used
Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Fabrazyme. It is used as an intravenous infusion of 1 mg per kilogram body weight given once every two weeks. The starting infusion rate should be no more than 0.25 mg per minute (15 mg per hour) to reduce the risk of infusion-related side effects. The infusion rate may be increased gradually with further infusions. One study has looked at what happens to Fabrazyme when given to children and suggested that Fabrazyme could be used in children between eight and 16 years of age at the same dose. Patients who have severe kidney damage may have a weaker response to treatment. Fabrazyme is intended for long-term use.
How it works
Fabrazyme is an enzyme replacement therapy. Enzyme replacement therapies provide patients with the enzyme they are lacking. Fabrazyme is designed to replace the human enzyme alpha-galactosidase A, which people with Fabry disease are lacking. The active substance in Fabrazyme, agalsidase beta, is a copy of the human enzyme, produced by a method known as ‘recombinant DNA technology’: it is made by a cell that has received a gene (DNA), which makes it able to produce the enzyme. The replacement enzyme helps to break down GL-3 and stops it building up (accumulating) in the patient’s cells.
Active substance: agalsidase beta
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