Fabry Disease

Active Ingredient: Agalsidase alfa

Indication for Agalsidase alfa

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old)

Agalsidase alfa is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease (α-galactosidase A deficiency).

For this indication, competent medicine agencies globally authorize below treatments:

0.2 mg/kg every other week

Route of admnistration

Intravenous

Defined daily dose

0.2 - 0.2 mg per kg of body weight

Dosage regimen

From 0.2 To 0.2 mg per kg of body weight once every 14 day(s)

Detailed description

The safety and efficacy of agalsidase alfa in children aged 0-6 years has not yet been established.

In clinical studies of children (7-18 years) who received agalsidase alfa 0.2 mg/kg every other week, no unexpected safety issues were encountered.

Dosage considerations

Intravenous infusion over 40 minutes.

Active ingredient

Agalsidase alfa

Agalsidase alfa catalyses the hydrolysis of Gb3, cleaving a terminal galactose residue from the molecule. Treatment with the enzyme has been shown to reduce accumulation of Gb3 in many cell types including endothelial and parenchymal cells. Agalsidase alfa has been produced in a human cell line to provide for a human glycosylation profile that can influence uptake by mannose-6-phosphate receptors on the surface of target cells.

Read more about Agalsidase alfa

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