==== Reference: Usmani SS, Bedi G, Samuel JS, Singh S, Kalra S, Kumar P, et al. (2017) THPdb: Database of FDA-approved peptide and protein therapeutics. PLoS ONE 12(7) e0181748.====

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Th1128 details
Primary information
ID1602
ThPP IDTh1128
Therapeutic Peptide/Protein NameVelaglucerase alfa
SequenceARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRRME view full sequnce in fasta
Functional ClassificationIa
Molecular Weight63000
Chemical FormulaC2532H3850N672O711S16
Isoelectric PointN.A.
HydrophobicityN.A.
Melting Point (℃)N.A.
Half Life11-12 Minutes
DescriptionGene-activated human recombinant glucocerebrosidase. It is used to treat 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.
Indication/DiseaseVelaglucerase 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.
PharmacodynamicsN.A.
Mechanism of ActionVelaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.
ToxicityN.A.
MetabolismN.A.
AbsorptionN.A.
Volume of DistributionThe mean volume of distribution at steady state ranges from 82 to 108 mL/kg (8.2% to 10.8% of body weight).
ClearanceMean clearance ranges from 6.72 to 7.56 mL/min/kg.
CategoriesEnzymes
Patents NumberUS7138262
Date of Issue22/11/10
Date of Expiry19/08/24
Drug InteractionN.A.
TargetGlucosylceramidase
Information of corresponding available drug in the market
Brand NameVPRIV
CompanyShire
Brand DiscriptionThe active ingredient of VPRIV is velaglucerase alfa, which is produced by gene activation technology in a human fibroblast cell line. Velaglucerase alfa is a glycoprotein of 497 amino acids; with a molecular weight of approximately 63 kDa. Velaglucerase alfa has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase. Velaglucerase alfa contains 5 potential N-linked glycosylation sites; four of these sites are occupied by glycan chains. Velaglucerase alfa is manufactured to contain predominantly high mannose-type N-linked glycan chains. The high mannose type N-linked glycan chains are specifically recognized and internalized via the mannose receptor present on the surface on macrophages, the cells that accumulate glucocerebroside in Gaucher disease. Velaglucerase alfa catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide in the lysosome. VPRIV is dosed by Units/kg, where one Unit of enzyme activity is defined as the quantity of enzyme required to convert one micromole of p-nitrophenyl β-D-glucopyranoside to pnitrophenol per minute at 37°C.
Prescribed forlong-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.
Chemical NameN.A.
Formulationvelaglucerase alfa (400 Units), citric acid, monohydrate (5.04 mg), polysorbate 20(0.44 mg), sodium citrate, dihydrate (51.76 mg), sucrose (200 mg)
Physcial AppearanceVPRIV is supplied as a sterile, preservative free, lyophilized powder
Route of AdministrationIntravenous infusion
Recommended DosageThe recommended dose is 60 Units/kg administered every other week as a 60-minute Intravenous infusion.
ContraindicationN.A.
Side EffectsHeadache, Dizziness, Abdominal pain, Nausea, Back pain, Joint pain (knee), Upper respiratory tract infection, Activated partial thromboplastin time prolonged, Infusion-related reaction*, Pyrexia, Asthenia/Fatigue
Useful Linkhttp://www.rxlist.com/vpriv-drug.htm http://www.vpriv.com/ http://www.drugs.com/vpriv.html
PubMed ID25501675, 24612151, 24581483, 24411065, 23430813, 20336596, 24630271
3-D StructureTh1128 (View) or (Download)