HOW ERT WORKS
ERT REPLACES OR SUPPLEMENTS DEFICIENT GLUCOCEREBROSIDASE2,3
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Glucocerebrosidase deficiency causes the build-up of lipids in the lysosomes, leading to the formation of Gaucher cells.4
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Over time, these Gaucher cells infiltrate various organs, particularly the spleen and liver, resulting in a progressive, heterogenous disease.4
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ERT is an infusion that replaces or supplements the deficient glucocerebrosidase enzyme.2,3
ERT is a First-line Treatment
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ERT is a first-line treatment for Gaucher disease and has been used for many years.2
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ERTs address the underlying enzyme deficiency2,3
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Treatment with ERTs has been used for visceral debulking (reducing liver and spleen volumes) and improving hemoglobin and platelet counts.2,3
HOW VPRIV WORKS
VPRIV, AN ERT
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VPRIV is a lysosomal glucocerebroside-specific enzyme indicated for long-term ERT for patients with type 1 Gaucher disease.1
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VPRIV was studied in the largest clinical trial program of an ERT for type 1 Gaucher disease.1,5
– VPRIV has been studied in up to 99 patients across three clinical trials.1
VPRIV’s Mechanism of Action (MOA)
VPRIV is Specifically Designed to Match And Replace the Natural Human Enzyme1,6
It is produced using gene-activation technology in a human cell line. It has the same amino acid sequence as the naturally occurring
human enzyme, glucocerebrosidase.1
VPRIV’s glycosylation pattern is intended to facilitate targeted uptake into macrophages*1,6–8
Gaucher Cell
Gaucher cells are swollen due to the accumulation of a fatty substance known as glucocerebroside.9,10
VPRIV ACTION
VPRIV is designed to bind to, and be absorbed into, the cell. Once inside the cell, VPRIV breaks down glucocerebroside.1
resulting Cell
Like the naturally occurring human enzyme, VPRIV breaks down glucocerebroside, reducing the overall amount in the cell.1
*For illustration purposes only. In vitro test results do not necessarily correlate with clinical efficacy.
