VPRIV SUMMARY
The active ingredient of VPRIV is velaglucerase alfa, which is produced by gene activation technology in a human fibroblast cell line.
VPRIV (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.
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NEWS HIGHLIGHTS
Published Studies Related to Vpriv (Velaglucerase Alfa)
Velaglucerase alfa: a new option for Gaucher disease treatment. [2011.07]
Type 1 Gaucher disease (GD) results from inherited beta-glucocerebrosidase gene mutations, leading to anemia, thrombocytopenia, splenomegaly, hepatomegaly and skeletal disease...
Significant and continuous improvement in bone mineral density among type 1 Gaucher disease patients treated with velaglucerase alfa: 69-month experience, including dose reduction. [2011.06.15]
Since bone pathology is a major concern in type 1 Gaucher disease (GD1), we evaluated bone mineral density (BMD) in adults receiving velaglucerase alfa in the seminal Phase I/II and extension trial. Ten treatment-naive symptomatic patients with GD1 (four men, six women; median age 35years, range 18-62years) were included; of these, four patients were receiving bisphosphonates at enrollment...
Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease. [2011.01.15]
INTRODUCTION: Therapeutic goals have been described to monitor achievement, maintenance and continuity of therapeutic response in patients with type 1 Gaucher disease receiving enzyme replacement therapy.This is the first report of a cohort where all patients receiving ERT for type 1 Gaucher disease achieved all 5 of these long-term, therapeutic goals within 4 years of starting treatment and after >/= 2years dose reduction.
Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. [2010.06.10]
Enzyme replacement therapy is the standard of care for symptomatic Gaucher disease. Velaglucerase alfa is a human beta-glucocerebrosidase produced in a well-characterized human cell line...
Comparative therapeutic effects of velaglucerase alfa and imiglucerase in a Gaucher disease mouse model. [2010.05.20]
Gaucher disease type 1 is caused by the defective activity of the lysosomal enzyme, acid beta-glucosidase (GCase)...
Clinical Trials Related to Vpriv (Velaglucerase Alfa)
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease [Recruiting]
The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone
development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural
history and neurological status of children with Type 3 Gaucher Disease will be studied.
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease [Completed]
The purpose of this study is to evaluate the long-term safety of every other week dosing of
Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in
patients with type 1 Gaucher disease.
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease [Completed]
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes
based on the presence or absence of neurological symptoms and the severity of these
neurological symptoms. Patients with type 2 Gaucher disease present with acute neurological
deterioration, and those with type 3 disease typically display a more sub acute neurological
course. Type 1 Gaucher disease, the most common form accounting for more than 90% of all
Gaucher disease cases, does not involve the central nervous system.
The purpose of this clinical research study is to investigate the safety and effectiveness
of velaglucerase alfa in patients with type 3 Gaucher disease.
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease [Completed]
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The
disease has been classified into 3 clinical subtypes based on the presence or absence of
neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects
an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does
not involve the central nervous system. Patients with type 2 Gaucher disease present with
acute neurological deterioration, which leads to early death. Those with type 3 disease
typically display a more sub-acute neurological course, with later onset and slower
progression.
The primary objective of this study is to evaluate the safety of every other week dosing of
velaglucerase alfa in Japanese patients with Gaucher disease.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type
1 Gaucher disease.
Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease [Completed]
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1
Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1
Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher
disease present with acute neurological deterioration, which leads to early death. Those
with Type 3 disease typically display a more sub-acute neurological course, with later onset
and slower progression.
The primary objective of this study is to evaluate the long-term safety of every other week
(EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed
study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type
1 Gaucher disease.
Reports of Suspected Vpriv (Velaglucerase Alfa) Side Effects
Infusion Related Reaction (9),
Death (6),
Chills (4),
Pyrexia (4),
Depression (4),
Anxiety (4),
Headache (3),
Hyperhidrosis (3),
Throat Tightness (3),
Lower Respiratory Tract Infection (3), more >>
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Page last updated: 2011-12-09
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