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Th1131 details
Primary information
ID1608
ThPP IDTh1131
Therapeutic Peptide/Protein NameTaliglucerase alfa
SequenceEFARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRR view full sequnce in fasta
Functional ClassificationIa
Molecular Weight56637.9397
Chemical FormulaC2580H3918N680O727S17
Isoelectric Point10.54
HydrophobicityN.A.
Melting Point (℃)N.A.
Half LifeN.A.
DescriptionRecombinant human glucocerebrosidase (a lysosomal enzyme) . Elelyso used in patients with type 1 Gaucher's disease
Indication/DiseaseFor the treatment of adult Type 1 Gaucher disease.
PharmacodynamicsPatient's with Type 1 Gaucher disease have a long-term deficiency in the enzyme, glucocerebrosidase. Taliglucerase alfa is a modified form of glucocerebrosidase and is provided to counter this enzyme deficiency, resulting in smaller liver and spleen size, and improved thrombocytopenia and anemia.
Mechanism of ActionTaliglucerase alfa is different from human glucocerebrosidase by two amino acids at the N terminal and up to 7 amino acids at the C terminal. This recombinant enzyme allows the hydrolysis reaction of glucocerebroside to glucose and ceramide that naturally occurs in healthy individuals.
ToxicityThe most common toxic reaction seen was infusion reactions such as urticaria, arthralgia, headache, and chest pain due to IV administration.
MetabolismMetabolism was not determined.
AbsorptionTaliglucerase alfa is administered IV so absorption is 100%.
Volume of DistributionThe steady state volume of distribution is between 7.30 to 11.7 L.
ClearanceThe systemic clearance was approximately 30 L/hr and 20 L/hr for 30 and 60 units/kg, respectively.
CategoriesEnzymes
Patents NumberN.A.
Date of IssueN.A.
Date of ExpiryN.A.
Drug InteractionN.A.
TargetGlucocerebroside
Information of corresponding available drug in the market
Brand NameElelyso
CompanyPfizer
Brand DiscriptionTaliglucerase alfa, a hydrolytic lysosomal glucocerebroside-specific enzyme for Intravenous infusion, is a recombinant active form of the lysosomal enzyme, β-glucocerebrosidase, which is expressed in genetically modified carrot plant root cells cultured in a disposable bioreactor system (ProCellEx). β-Glucocerebrosidase (β-D-glucosyl-Nacylsphingosine glucohydrolase, E.C. 3.2.1.45) is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide. ELELYSO is produced by recombinant DNA technology using plant cell culture (carrot). Purified taliglucerase alfa is a monomeric glycoprotein containing 4 N-linked glycosylation sites (Mr = 60,800). Taliglucerase alfa differs from native human glucocerebrosidase by two amino acids at the N terminal and up to 7 amino acids at the C terminal. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. These mannose-terminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in Gaucher disease.
Prescribed forlong-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
Chemical NameN.A.
FormulationThe quantitative composition of each 200 unit vial is D-mannitol (206.7 mg), polysorbate 80 (0.56 mg), sodium citrate (30.4 mg), and taliglucerase alfa (212 units). Citric acid may be added to adjust the pH at the time of manufacture.
Physcial AppearanceELELYSO is supplied as a sterile, non-pyrogenic, lyophilized powder.
Route of AdministrationIntravenous infusion
Recommended Dosage60 units per kg of body weight
ContraindicationN.A.
Side Effectspruritus, flushing, headache, arthralgia, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea, and rash.
Useful Linkhttp://www.rxlist.com/elelyso-drug.htm http://www.elelyso.com/ http://www.drugs.com/elelyso.html
PubMed ID23430813, 25453586, 24950666, 24630271, 23199589, 21900191
3-D StructureTh1131 (View) or (Download)