Replagal is a solution for infusion into the body containing the active substance Agalsidase alfa.
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, alpha-galactosidase A.
This enzyme normally breaks down a fatty substance called globotriaosylceramide ( Gb3 ). If the enzyme is not present, Gb3 cannot be broken down and it builds up in the 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.

Replagal is used as an intravenous infusion of 0.2 mg/kg body weight over 40 minutes given once every 2 weeks.
Replagal could be used in children between 7 and 18 years of age at the same dose.
Patients who have severe kidney damage may have a weaker response to treatment.
Replagal is intended for long-term use.

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 people with Fabry disease are lacking. The active substance in Replagal, Agalsidase alfa, is a copy of the human enzyme, produced by a method known as recombinant DNA technology.
The replacement enzyme helps to break down the Gb3 and stops it building up in the cells.

Replagal has been examined in two clinical studies, involving a total of 40 male patients. Replagal was compared with a placebo and its effect on pain was measured in one study and its effect on clearing Gb3 from the left ventricle was studied in the other.
A further study was also carried out in 15 female patients.
Replagal has also been studied in 24 children aged between 6 and a half and 18 years of age.

After 6 months of therapy Replagal significantly reduced pain in patients when compared to placebo treated patients.
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.
These results suggest the symptoms of the disease improving or the disease becoming stable.
The effects in female patients were shown to be comparable to the results seen in male patients. Children who received 6-month treatment with Replagal had no increase in heart, and the levels of Gb3 in their blood was reduced.

During the studies the most common side effects ( seen in more than 1 patient in 10 ) are caused by the infusion rather than the medicine. These reactions are mainly chills, headache, nausea, pyrexia, flushing and fatigue, and are rarely severe.
Other very common side effects were pain and discomfort.
Side effects reported in children are similar to those seen in adult patients.

Patients who receive Replagal can develop antibodies.

Replagal should not be used in people who may be hypersensitive to Agalsidase alfa or any of the other ingredients.

Source: EMEA, 2007

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