What is Replagal?
Replagal is a solution for infusion, which contains the active substance agalsidase alfa.
What is Replagal used for?
Replagal is used to treat patients with Fabry disease, a rare inherited condition.
Patients suffering from this disease have a deficiency of the enzyme alpha-galactosidase A. This enzyme normally breaks down the lipid globotriaosylceramide (Gb3). If this enzyme is missing, the Gb3 cannot be broken down and accumulates in cells, for example in those cells. kidney.
Patients with Fabry disease can have a wide range of symptoms, which include serious ailments, such as kidney failure, heart problems and stroke.
As the number of patients with Fabry disease is low, this disease is considered 'rare' and Replagal was designated an 'orphan medicine' (a medicine used to treat rare diseases) on 8 August 2000.
The medicine can only be obtained with a prescription.
How is Replagal used?
Replagal must be administered under the supervision of a doctor who specializes in the treatment of Fabry disease or other inherited metabolic disorders. It is given as an intravenous infusion of 0.2 mg / kg body weight for 40 minutes every 2 weeks. The effect of Replagal given to children has been examined in some studies and it has therefore been suggested that Replagal can be used in children from 7 to 18 years at the same dose. Patients who have severe kidney problems have a lower response to treatment. Replagal is intended for long-term use.
How does Replagal work?
Replagal is enzyme replacement therapy, which is a therapy that provides patients with the enzyme they lack. Replagal is intended to replace the human enzyme alpha-galactosidase A, which people with Fabry disease lack. The active substance in Replagal, agalsidase alfa, is a copy of the human enzyme produced by a method called 'recombinant DNA technology': the enzyme is made by a cell that has received a gene (DNA) which makes it capable of producing the enzyme. This replacement enzyme promotes the breakdown of Gb3 preventing its accumulation in the cells.
How has Replagal been studied?
Replagal has been studied in two clinical studies involving a total of 40 male patients. Replagal was compared with placebo (a dummy treatment); in one study its effect on pain was measured, while in another study its effect on the elimination of Gb3 from the left ventricle (myocardium) was examined. A study in 15 female patients (carriers) was also carried out.
Replagal has also been studied in 24 children between the ages of 6 and a half to 18 years.
What benefit has Replagal shown during the studies?
After 6 months of therapy, Replagal significantly reduced pain in the treated patients compared to those receiving placebo (a dummy treatment). Replagal produced an average reduction in left ventricular mass of 11.5 g, while patients treated with placebo experienced an increase of 21.8 g. These results indicate that the symptoms of the disease improve or that the disease remains stable. In women, the effects were comparable to the results seen in men. The infants who received 6 months of Replagal treatment did not show an increase in cardiac mass and blood Gb3 levels had decreased.
What is the risk associated with Replagal?
The most common side effects (seen in more than one in 10 patients during the studies) are caused by the infusion rather than the medicine itself. These are mainly chills, headache, nausea, pyrexia (fever), flushing of the face and fatigue (tiredness), which are generally not serious. Other very common side effects include pain and discomfort. Side effects reported in children are similar to those seen in adults. For the full list of side effects reported with Replagal, please see refer to the package leaflet.
Patients taking Replagal can develop antibodies (proteins produced as a reaction to Replagal, which can impair treatment).
Replagal must not be used in people who may be hypersensitive (allergic) to agalsidase alfa or other ingredients of the medicine.
Why has Replagal been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that for patients with Fabry disease, treatment with Replagal can produce clinical benefits in the long term. The CHMP decided that Replagal's benefits are greater than its risks and recommended that it be given a Marketing Authorization for the medicine.
Replagal was authorized "in exceptional circumstances", as it was not possible to obtain more detailed information on the medicine as it is used to treat a rare disease. The European Medicines Agency (EMEA) reviews the new information available annually and, if necessary, this summary will be updated.
What information is still awaited for Replagal?
The company that makes Replagal will carry out further studies on the medicine, mainly to obtain results from 5 years of therapy, other doses, maintenance doses and studies in children.
More information about Replagal
On 3 August 2001 the European Commission granted TKT Europe AB a "Marketing Authorization" for Replagal, valid throughout the European Union. The "Marketing Authorization" was renewed on 3 August 2006. For the Replagal's orphan status registration, click here.
For the full version of Replagal's EPAR, click here.
Last update of this summary: 02-2007
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