Detailed description page of ThPDB2

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10636 details
Primary information
ID10636
Therapeutic IDTh1131
Protein NameTaliglucerase alfa
Sequence>Th1131_Taliglucerase_alfa EFARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRR
Molecular Weight56637.94
Chemical FormulaC2580H3918N680O727S17
Isoelectric Point10.54
HydrophobicityNA
Melting pointNA
Half-lifebetween 18.9 to 28.7 min.
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%.
The 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 NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetGlucocerebroside
Brand NameElelyso
CompanyPfizer
Brand DescriptionPfizer
Prescribed Forlong-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
Chemical NameNA
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.
Physical Appearance ELELYSO is supplied as a sterile, non-pyrogenic, lyophilized powder.
Route of AdministrationIntravenous infusion
Recommended Dosage60 units per kg of body weight
ContraindicationNA
Side Effectspruritus, flushing, headache, arthralgia, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea, and rash.
Useful Link 1Link
Useful Link 2NA
RemarksNA