Velaglucerase alfa

Identification

Summary

Velaglucerase alfa is an enzyme replacement therapy used for the long-term treatment of for pediatric and adult patients with type 1 Gaucher disease.

Brand Names
Vpriv
Generic Name
Velaglucerase alfa
DrugBank Accession Number
DB06720
Background

Velaglucerase alfa is a gene-activated human recombinant glucocerebrosidase used for the treatment of Type 1 Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase. Additionally, Velaglucerase alfa has also been investigated for use in Type 3 Gaucher disease.

Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
Protein Chemical Formula
C2532H3850N672O711S16
Protein Average Weight
63000.0 Da (approximate)
Sequences
>"VPRIV" Sequence
ARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANH
TGTGLLLTLQPEQKFQKVKGFGGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIR
VPMASCDFSIRTYTYADTPDDFQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWT
SPTWLKTNGAVNGKGSLKGQPGDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGL
LSGYPFQCLGFTPEHQRDFIARDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPE
AAKYVHGIAVHWYLDFLAPAKATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRG
MQYSHSIITNLLYHVVGWTDWNLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHL
GHFSKFIPEGSQRVGLVASQKNDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFL
ETISPGYSIHTYLWRRQ
Download FASTA Format
Synonyms
  • GA-GCB
  • Velaglucerasa alfa
  • Velaglucerase alfa

Pharmacology

Indication

Velaglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease.

Pharmacology
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Associated Conditions
Contraindications & Blackbox Warnings
Contraindications
Avoid life-threatening adverse drug events
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Pharmacodynamics

Not Available

Mechanism of action

Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.

TargetActionsOrganism
UGlucosylceramidaseNot AvailableHumans
Absorption

Not Available

Volume of distribution

The mean volume of distribution at steady state ranges from 82 to 108 mL/kg (8.2% to 10.8% of body weight).

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

11-12 minutes.

Clearance

Mean clearance ranges from 6.72 to 7.56 mL/min/kg.

Adverse Effects
Adverseeffects
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Toxicity

Not Available

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
No interactions found.
Food Interactions
No interactions found.

Products

Products2
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International/Other Brands
Imiglucerase (Genzyme Corporation)
Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
VprivInjection, powder, for solution400 UIntravenousShire2016-09-08Not applicableEU flag
VprivPowder, for solution400 unit / vialIntravenousTakeda2010-11-22Not applicableCanada flag
VprivInjection, powder, for solution400 UIntravenousShire2016-09-08Not applicableEU flag
VprivInjection, powder, for solution400 UIntravenousShire2016-09-08Not applicableEU flag
VprivInjection, powder, lyophilized, for solution2.5 mg/1mLIntravenousTakeda Pharmaceuticals America, Inc.2010-02-26Not applicableUS flag

Categories

ATC Codes
A16AB10 — Velaglucerase alfa
Drug Categories
Chemical TaxonomyProvided by Classyfire
Description
Not Available
Kingdom
Organic Compounds
Super Class
Organic Acids
Class
Carboxylic Acids and Derivatives
Sub Class
Amino Acids, Peptides, and Analogues
Direct Parent
Peptides
Alternative Parents
Not Available
Substituents
Not Available
Molecular Framework
Not Available
External Descriptors
Not Available
Affected organisms
Not Available

Chemical Identifiers

UNII
23HYE36B0I
CAS number
884604-91-5

References

General References
  1. Pastores GM: Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease. Curr Opin Investig Drugs. 2010 Apr;11(4):472-8. [Article]
  2. Vairo F, Netto C, Dorneles A, Mittelstadt S, Wilke M, Doneda D, Michelin K, Ribeiro CB, Quevedo A, Vieira T, Nalin T, Lueska S, Schwartz IV: Enzyme Replacement Therapy in a Patient with Gaucher Disease Type III: A Paradigmatic Case Showing Severe Adverse Reactions Started a Long Time After the Beginning of Treatment. JIMD Rep. 2013;11:1-6. doi: 10.1007/8904_2013_214. Epub 2013 Feb 21. [Article]
UniProt
P04062
KEGG Drug
D09029
PubChem Substance
347910363
RxNav
901805
ChEMBL
CHEMBL1201865
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
PDRhealth
PDRhealth Drug Page
Wikipedia
Velaglucerase_alfa
FDA label
Download (162 KB)
MSDS
Download (568 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
4Active Not RecruitingTreatmentGaucher Disease, Type 11
4CompletedTreatmentGaucher's Disease1
4CompletedTreatmentPrimary Disease1
4RecruitingTreatmentGaucher's Disease1
4WithdrawnTreatmentGaucher Disease, Type 1 / Gaucher Disease, Type 31
3CompletedTreatmentGaucher Disease, Type 13
3CompletedTreatmentGaucher's Disease2
2, 3CompletedTreatmentGaucher's Disease1
1, 2CompletedTreatmentGaucher Disease, Type 31
1, 2CompletedTreatmentGaucher's Disease1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
Injection, powder, for solutionIntravenous400 U
Injection, powder, lyophilized, for solutionIntravenous2.5 mg/1mL
Powder, for solutionIntravenous400 unit / vial
Powder, for solutionIntravenous; Parenteral200 U
Injection, powder, lyophilized, for solutionIntravenous10 mg
Injection, powder, for solutionParenteral
Prices
Not Available
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)Region
US7138262No2006-11-212020-08-18US flag

Properties

State
Solid
Experimental Properties
Not Available

Targets

Drugtargets2
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Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Receptor binding
Specific Function
Not Available
Gene Name
GBA
Uniprot ID
P04062
Uniprot Name
Glucosylceramidase
Molecular Weight
59715.745 Da
References
  1. Pastores GM: Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease. Curr Opin Investig Drugs. 2010 Apr;11(4):472-8. [Article]

Drug created on June 10, 2010 20:05 / Updated on September 19, 2021 19:53