Active Substance: agalsidase alfa
Common Name: agalsidase alfa
ATC Code: A16AB03
Marketing Authorisation Holder: Shire Human Genetic Therapies AB
Active Substance: agalsidase alfa
Authorisation Date: 2001-08-03
Therapeutic Area: Fabry Disease
Pharmacotherapeutic Group: Other alimentary-tract and metabolism products
Replagal is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of Fabry disease (α-galactosidase-A deficiency).
What is Replagal?
Replagal is medicine that contains the active substance agalsidase alfa. It is available as a concentrate to be made into solution for infusion (drip) into a vein.
What is Replagal used for?
Replagal 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 (Gb3 or GL-3). If the enzyme is not present, Gb3 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.
The medicine can only be obtained with a prescription.
How is Replagal used?
Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Replagal.
Replagal is given once every two weeks as an infusion of 0.2 mg/kg body weight over 40 minutes. It is intended for long-term use.
How does Replagal work?
Replagal is an enzyme-replacement therapy. Enzyme-replacement therapy provides patients with the enzyme they are lacking. Replagal is designed to replace the human enzyme alpha-galactosidase A, which is lacking in people with Fabry disease. The active substance in Replagal, agalsidase alfa, 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 the Gb3 and stops it building up in the cells.
How has Replagal been studied?
Replagal has been compared with placebo (a dummy treatment) in two main studies involving a total of 40 male patients. The first study measured the effects of Replagal on pain while the second study measured its effect on the mass of the left ventricle (heart muscle), a measure of the amount of Gb3 in the heart cells. A further study was also carried out in 15 female patients. Replagal has also been studied in 24 children aged between six and a half and 18 years of age.
What benefit has Replagal shown during the studies?
After six months of treatment, Replagal significantly reduced pain in patients compared with placebo. Replagal reduced left ventricle mass by an average of 11.5 g while patients receiving placebo had an increase in left ventricular mass of 21.8 g. The effects in female patients were comparable to those in male patients. Children who received Replagal for 6 months had no increase in heart mass, and the levels of Gb3 in their blood were reduced.
What is the risk associated with Replagal?
The most common side effects with Replagal (seen in more than 1 patient in 10) are infusion-associated reactions. These include chills, headache, nausea, pyrexia (fever), pain and discomfort, flushing and fatigue (tiredness), and are rarely severe. For the full list of all side effects reported with Replagal, see the package leaflet.
Replagal must not be used in people who are hypersensitive (allergic) to agalsidase alfa or any of the other ingredients.
Why has Replagal been approved?
The CHMP decided that for patients with Fabry disease treatment with Replagal might provide long-term clinical benefits. The CHMP decided that Replagal’s benefits are greater than its risks and recommended that it be given marketing authorisation.
Replagal has been authorised under ‘exceptional circumstances’. This means that because the disease is rare, it has not been possible to obtain complete information on the medicine. Every year, the European Medicines Agency will review any new information that may have become available and this summary will be updated as necessary.
What information is still awaited for Replagal?
The company that makes Replagal will carry further studies with the medicine, looking in particular at the results of five-year treatment, other dosages, maintenance dosages, and studies in children.
Other information about Replagal
The European Commission granted a marketing authorisation valid throughout the European Union for Replagal on 3 August 2001.
For more information about treatment with Replagal, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
Source: European Medicines Agency
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