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HOW ERT WORKS & VPRIV MOA

VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for first-line treatment for patients with type 1 Gaucher disease1,2
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Female patient
HOW ERT WORKS

    ERT replaces or supplements deficient glucocerebrosidase.2,3

  • Glucocerebrosidase deficiency causes the build-up of lipids in the lysosomes, leading to the formation of Gaucher cells4
  • Over time, these Gaucher cells infiltrate various organs, particularly the spleen and liver, resulting in a progressive, heterogeneous disease4
  • ERT is an infusion that replaces or supplements the deficient glucocerebrosidase enzyme2,3
ERT IS A FIRST-LINE TREATMENT
  • ERT is a first-line treatment for Gaucher disease that has been used for many years2
  • ERTs address the underlying enzyme deficiency2,3
  • Treatment with ERTs has been used for visceral debulking (reducing spleen and liver volumes) and improving hemoglobin and platelet counts2,3
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VPRIV MECHANISM OF ACTION (MOA)

    VPRIV, an ERT

  • VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term ERT for patients with type 1 Gaucher disease1
  • VPRIV’s safety and efficacy were evaluated in the largest clinical trial program of an ERT for type 1 Gaucher disease; VPRIV was studied across three clinical trials (n=99; aged ≥4 years)1,5,6

    VPRIV is specifically designed to match and replace the natural human enzyme.1,7

  • VPRIV is produced using gene-activation technology in a human cell line1
  • VPRIV has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase1
Glucocerebrosidase

Glucocerebrosidase

VPRIV

VPRIV

VPRIV’s glycosylation pattern is intended to facilitate targeted uptake into macrophages.*1,7–9

VPRIV Mechanism of Action Video

Gaucher Cell
1
GAUCHER CELL

Gaucher cells are swollen due
to the accumulation of a fatty substance known as glucocerebroside.10,11

VPRIV action
2
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
3
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

VPRIV HCP Brochure

Summary of VPRIV, an ERT for type 1 Gaucher disease

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.