Products
Velaglucerase alfa is commercially available as a powder for the preparation of an infusion solution (Vpriv). It has been approved in many countries since 2011.
Structure and properties
Velaglucerase alfa is an enzyme and glycoprotein with 497 amino acids and the same sequence as natural glucocerebrosidase. Glycosylation with mannose results in velaglucerase alfa being taken up predominantly into macrophages.
Effects
Velaglucerase alfa (ATC A16AB10) is an analog of the enzyme beta-glucocerebrosidase. This enzyme hydrolyzes glucocerebroside into glucose and ceramide. Gaucher disease is characterized by a deficiency of this lysosomal enzyme. This leads to accumulation of glucocerebroside in cells, mainly in macrophages.
Indications
For long-term treatment of patients with Gaucher disease type 1.
Dosage
According to the SmPC. The drug is administered as an intravenous infusion every two weeks.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Interactions
There are no known drug-drug interactions.
Adverse effects
The most common possible adverse effects include:
