This is a summary of the European public assessment report (EPAR) for Fabrazyme. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Fabrazyme.
What is Fabrazyme?
Fabrazyme is a medicine that contains the active substance agalsidase beta. It is available as a powder to be made into a solution for infusion (drip) into a vein.
What is Fabrazyme 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
People with Fabry disease may have a wide range of signs and symptoms, including severe conditions such as kidney failure, heart problems and stroke.
The medicine can only be obtained with a prescription.
How is Fabrazyme used?
Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Fabrazyme.
Fabrazyme is given once every two weeks as an infusion of 1 mg per kilogram body weight. The starting infusion rate should be no more than 0.25 mg per minute (15 mg per hour) to reduce the risk of
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Fabrazyme is intended for
How does Fabrazyme work?
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 is lacking in patients with Fabry disease. 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 cells that have received a gene (DNA), which makes them able to produce the enzyme. The replacement enzyme helps to break down
How has Fabrazyme been studied?
Fabrazyme has been investigated in three studies involving a total of 73 adults. In the main study, Fabrazyme was compared with placebo (a dummy treatment) in 58 patients. The study looked at the effects of the medicine on clearing
What benefit has Fabrazyme shown during the studies?
In the main study, Fabrazyme produced a highly significant and almost complete clearance of
Children treated with Fabrazyme also had decreases in levels of
What is the risk associated with Fabrazyme?
The most common side effects with Fabrazyme (seen in more than 1 patient in 10) are caused by the infusion rather than the medicine. These include fever, chills, headache, paraesthesia (abnormal sensations like pins and needles), nausea (feeling sick), vomiting and feeling cold. For the full list of all side effects reported with Fabrazyme, see the package leaflet.
Fabrazyme must not be used in people who are hypersensitive (allergic) to agalsidase beta or any of the other ingredients.
Why has Fabrazyme been approved?
The CHMP decided that, for patients with Fabry disease, treatment with Fabrazyme might provide long- term clinical benefits. The CHMP decided that Fabrazyme’s benefits are greater than its risks and recommended that it be given marketing authorisation.
Other information about Fabrazyme
The European Commission granted a marketing authorisation valid throughout the European Union for Fabrazyme on 3 August 2001.
The full EPAR for Fabrazyme can be found on the Agency’s website: ema.europa.eu/Find medicine/Human medicines/European Public Assessment Reports. For more information about
treatment with Fabrazyme, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
This summary was last updated in