Velaglucerase Alfa Drug Aliquot | Anti-Glucosylceramidade | Therapeutic Enzyme

Aliquoted from the original reference drug | Now available for research use

Aliquots of the reference drug Velaglucerase alfa are now available as research consumables.

Velaglucerase alfa drug aliquots can be used for in vitro or in vivo experiments. For biosimilar development, different batches of Velaglucerase alfa drug can be ordered at the same time. Several other reference drugs are also available.

The drug aliquots are generated by aliquoting (dividing and repackaging) the EU-licensed reference drug.

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Vpriv® / Velaglucerase alfa

Drug nameVpriv®
INNVelaglucerase alfa
API typeVelaglucerase alfa is produced in an HT-1080 human fibroblast cell line by recombinant DNA technology.
Pharmacotherapeutic groupOther alimentary tract and metabolism products, enzymes
ATC codeA16AB10
Target of antibody
General functionVelaglucerase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease.
Short description
Pharmacodynamic properties
(Mechanism of action; Source EMA document)
Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, the enzyme that catalyses 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.
Pharmacodynamic properties (Pharmacodynamic effects; Source EMA document)Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of glucocerebroside primarily in macrophages, giving rise to foam cells or "Gaucher cells". In this lysosomal storage disorder (LSD), clinical features are reflective of the distribution of Gaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. The accumulation of glucocerebroside in the liver and spleen leads to organomegaly. Bone involvement results in skeletal abnormalities and deformities as well as bone pain crises. Deposits in the bone marrow and splenic sequestration lead to clinically significant anaemia and thrombocytopenia.
Original license holder
Marketing authorisation numbersEU/1/10/646/002
Marketing authorisation holderTakeda Pharmaceuticals International AG Ireland Branch Block 3 Miesian Plaza 50 – 58 Baggot Street Lower Dublin 2 Ireland
Name of the manufacturer of the biological active substanceCell Bank storage and Drug Substance Manufacture
Shire Human Genetic Therapies, Inc
205 Alewife Brook Parkway, Cambridge, Massachusetts 02138
Drug Substance Manufacture
Shire Human Genetic Therapies, Inc
400 Shire Way, Lexington, Massachusetts 02421
Name and address of the manufacturer(s) responsible for batch releaseShire Pharmaceuticals Ireland Limited Block 2 & 3 Miesian Plaza 50 – 58 Baggot Street Lower Dublin 2 Ireland
Max shelf life3 years
Storage conditions2°C-8°C
List of excipientsSucrose - Sodium citrate dihydrate (E331) - Citric acid monohydrate (E330) - Polysorbate 20

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Evidentic offers multiple batches of original drug aliquots in low binding Eppendorf tubes for research use only. We provide licence-free drug aliquots with long, short or even “negative shelf life” by storing the products at recommended temperatures (either 2-8°C or -80°C) and ensuring fully functional molecules for research purposes.