Gaucher disease type 1

Active Ingredient: Velaglucerase alfa

Indication for Velaglucerase alfa

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

Velaglucerase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease.

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

15-60 IU/kg once every other week

Route of admnistration

Intravenous

Defined daily dose

60 - 60 [iU] per kg of body weight

Dosage regimen

From 60 To 60 [iU] per kg of body weight once every 14 day(s)

Detailed description

The recommended dose is 60 Units/kg administered every other week.

Dose adjustments can be made on an individual basis based on achievement and maintenance of therapeutic goals. Clinical studies have evaluated doses ranging from 15 to 60 Units/kg every other week. Doses higher than 60 Units/kg have not been studied.

Patients currently treated with imiglucerase enzyme replacement therapy for type 1 Gaucher disease may be switched to velaglucerase alfa, using the same dose and frequency.

Dosage considerations

To be administered as a 60-minute intravenous infusion.

Active ingredient

Velaglucerase alfa

Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, the enzyme that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide in the lysosome, reducing the amount of accumulated glucocerebroside and correcting the pathophysiology of Gaucher disease. Velaglucerase alfa increases haemoglobin concentration and platelet counts and reduces liver and spleen volumes in patients with type 1 Gaucher disease.

Read more about Velaglucerase alfa

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