Velaglucerase alfa

Identification

Name
Velaglucerase alfa
Accession Number
DB06720  (DB05543)
Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
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 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
  • Imiglucerase
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
VprivPowder, for solution400 unitIntravenousShire Human Genetic Therapies, Inc.2010-11-22Not applicableCanada
VprivInjection, powder, for solution400 UIntravenousShire2010-08-26Not applicableEu
VprivInjection, powder, for solution400 UIntravenousShire2010-08-26Not applicableEu
VprivInjection, powder, lyophilized, for solution2.5 mg/mLIntravenousShire2010-02-26Not applicableUs
VprivInjection, powder, for solution400 UIntravenousShire2010-08-26Not applicableEu
International/Other Brands
Imiglucerase (Genzyme Corporation) / VPRIV
Categories
UNII
23HYE36B0I
CAS number
884604-91-5

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.

Structured Indications
Pharmacodynamics
Not Available
Mechanism of action

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

TargetActionsOrganism
UGlucosylceramidaseNot AvailableHuman
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.

Toxicity
Not Available
Affected organisms
Not Available
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
No interactions found.
Food Interactions
Not Available

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. [PubMed:20336596]
  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. [PubMed:23430813]
External Links
UniProt
P04062
KEGG Drug
D09029
PubChem Substance
347910363
ChEMBL
CHEMBL1201865
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
PDRhealth
PDRhealth Drug Page
Wikipedia
Velaglucerase_alfa
ATC Codes
A16AB10 — Velaglucerase alfa
AHFS Codes
  • 44:00.00 — Enzymes
FDA label
Download (162 KB)
MSDS
Download (568 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1, 2CompletedTreatmentGaucher Disease, Type 1 / Gaucher's Disease1
1, 2CompletedTreatmentGaucher Disease, Type 31
2, 3CompletedTreatmentGaucher Disease, Type 11
3CompletedTreatmentGaucher Disease, Type 13
3CompletedTreatmentGaucher's Disease2
4RecruitingTreatmentGaucher Disease Type 3 / Gaucher's Disease Type 11
4RecruitingTreatmentGaucher's Disease / Gaucher's Disease Type 11
Not AvailableApproved for MarketingNot AvailableGaucher Disease, Type 11

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage forms
FormRouteStrength
Injection, powder, for solutionIntravenous400 U
Injection, powder, lyophilized, for solutionIntravenous2.5 mg/mL
Powder, for solutionIntravenous400 unit
Prices
Not Available
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)
US7138262No2006-11-212020-08-18Us

Properties

State
Solid
Experimental Properties
Not Available

Taxonomy

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

Targets

Kind
Protein
Organism
Human
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. [PubMed:20336596]

Drug created on June 10, 2010 14:05 / Updated on January 14, 2018 10:04