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Identification
NameVelaglucerase alfa
Accession NumberDB06720
TypeBiotech
GroupsApproved, Investigational
Description

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.

Protein structureNo structure small
Protein chemical formulaC2532H3850N672O711S16
Protein average weight~63 kDa
Sequences
>"VPRIV" Sequence
ARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANH
TGTGLLLTLQPEQKFQKVKGFGGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIR
VPMASCDFSIRTYTYADTPDDFQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWT
SPTWLKTNGAVNGKGSLKGQPGDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGL
LSGYPFQCLGFTPEHQRDFIARDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPE
AAKYVHGIAVHWYLDFLAPAKATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRG
MQYSHSIITNLLYHVVGWTDWNLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHL
GHFSKFIPEGSQRVGLVASQKNDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFL
ETISPGYSIHTYLWRRQ
Download FASTA Format
Synonyms
SynonymLanguageCode
ImigluceraseNot AvailableNot Available
VPRIVNot AvailableNot Available
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
Vprivinjection, powder, lyophilized, for solution2.5 mg/mLintravenousShire US Manufacturing Inc.2010-02-26Not AvailableUs
Vprivpowder for solution400 unitintravenousShire Human Genetic Therapies IncNot AvailableNot AvailableCanada
Generic Prescription ProductsNot Available
Over the Counter ProductsNot Available
International Brands
NameCompany
ImigluceraseGenzyme Corporation
Brand mixturesNot Available
SaltsNot Available
Categories
CAS number884604-91-5
Taxonomy
DescriptionNot Available
KingdomOrganic Compounds
Super ClassOrganic Acids
ClassCarboxylic Acids and Derivatives
Sub ClassAmino Acids, Peptides, and Analogues
Direct ParentPeptides
Alternative ParentsNot Available
SubstituentsNot Available
Molecular FrameworkNot Available
External DescriptorsNot Available
Pharmacology
IndicationVelaglucerase 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.
PharmacodynamicsNot Available
Mechanism of actionVelaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.
AbsorptionNot 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 bindingNot Available
Metabolism
Route of eliminationNot Available
Half life11-12 minutes.
Clearance

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

ToxicityNot Available
Affected organismsNot Available
PathwaysNot Available
SNP Mediated EffectsNot Available
SNP Mediated Adverse Drug ReactionsNot Available
Pharmacoeconomics
ManufacturersNot Available
PackagersNot Available
Dosage forms
FormRouteStrength
Injection, powder, lyophilized, for solutionintravenous2.5 mg/mL
Powder for solutionintravenous400 unit
PricesNot Available
Patents
CountryPatent NumberApprovedExpires (estimated)
United States71382622006-11-212020-08-18
Properties
StateSolid
Experimental PropertiesNot Available
References
Synthesis ReferenceNot Available
General Reference
  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. Pubmed
  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 Feb 21. Pubmed
External Links
ATC CodesA16AB10
AHFS CodesNot Available
PDB EntriesNot Available
FDA labelDownload (162 KB)
MSDSDownload (568 KB)
Interactions
Drug InteractionsSearched, but no interactions found.
Food InteractionsNot Available

Targets

1. Glucosylceramidase

Kind: protein

Organism: Human

Pharmacological action: unknown

Components

Name UniProt ID Details
Glucosylceramidase P04062 Details

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. Pubmed

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Drug created on June 10, 2010 14:05 / Updated on April 26, 2013 12:10