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Drug

Velaglucerase alfa

Established medicine
ResearchOngoing clinical research

Also known as GA-GCB, VPRIV, Velaglucerasa alfa.

RxNorm901805UNII23HYE36B0I
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

Clinical trial2025-06-26Results posted · ClinicalTrials.gov

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

Clinical trial2022-02-01Results posted · ClinicalTrials.gov

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study

Clinical trial2022-01-01Completed · ClinicalTrials.gov

A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Clinical trial2021-06-28Results posted · ClinicalTrials.gov

An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy

Clinical trial2021-06-22Results posted · ClinicalTrials.gov

A Multicenter, Open-label Extension Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Clinical trial2021-06-14Results posted · ClinicalTrials.gov

A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease

Clinical trial2021-06-11Results posted · ClinicalTrials.gov

A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Clinical trial2021-06-10Results posted · ClinicalTrials.gov

A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease

Clinical trial2021-06-08Results posted · ClinicalTrials.gov

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

Clinical trial2021-03-18Completed · ClinicalTrials.gov

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Clinical trial2017-10-01Withdrawn · ClinicalTrials.gov

Accelerated approval: Vpriv (EMA)

Regulatory2010-08-26EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Velaglucerase alfa
Aliases & brands
GA-GCBVPRIVVelaglucerasa alfa
RxNorm CUI
901805
UNII
23HYE36B0I
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2010-08-26
Latest approval
2010-08-26
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2010-08-26Accelerated approval
Accelerated approval: Vpriv (EMA)

Indication: Vpriv is indicated for long-term enzyme-replacement therapy (ERT) in patients with type-1 Gaucher disease.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

11 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
11
registered trials across all phases
LATEST COMPLETION 2024
9
Late-stage (III+)
10
Completed
1
Discontinued
PHASE DISTRIBUTIONn = 11
Phase 1 / 22Phase 2 / 31Phase 34Phase 44

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study

CompletedPH IVShaare Zedek Medical Centern=152022

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

CompletedPH IVShaare Zedek Medical Centern=152021

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

CompletedPH IVShiren=212020

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

WithdrawnPH IVBaylor Research Institute2017

A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

CompletedPH IIITakedan=202024

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

CompletedPH IIITakedan=202024

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study

CompletedPH IVShaare Zedek Medical Centern=152022

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

CompletedPH IVShaare Zedek Medical Centern=152021

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

CompletedPH IVShiren=212020

A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease

CompletedPH I/IIShiren=72015

A Multicenter, Open-label Extension Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

CompletedPH IIIShiren=52014
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.