Velaglucerase alfa

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Information about Velaglucerase alfa

Velaglucerase (vel" a gloo' ser ase) alfa was the second recombinant form of glucocerebrosidase approved as therapy of Gaucher disease. Velaglucerase is produced in a human cell line using gene activation technology, resulting in production of a recombinant protein with the identical amino acid sequence as the native, human enzyme. The glucocerebrosidase producing cells are treated with enzymes that modify glycosylation and result in high-mannose type glycans which increase uptake of the velaglucerase by macrophages.

FDA approval information for Velaglucerase alfa

Velaglucerase was approved for use as enzyme replacement therapy of type 1 Gaucher disease in the United States in 2010. It is available as a lyophilized powder in single use vials of 400 Units under the brand name Vpriv. The typical initial dose is 60 Units/kg intravenously every 2 weeks.

Side effects of Velaglucerase alfa

Side effects are not common and usually mild, but can include local infusion reactions, fatigue, headache, dizziness, abdominal pain, nausea, back pain, joint pain and fever. Rare, but reported severe adverse reactions include hypersensitivity reactions and anaphylaxis.

genetic disorder agents

cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents


homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

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