Primary information |
---|
ID | 10636 |
Therapeutic ID | Th1131 |
Protein Name | Taliglucerase alfa |
Sequence | >Th1131_Taliglucerase_alfa
EFARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRR
|
Molecular Weight | 56637.94 |
Chemical Formula | C2580H3918N680O727S17 |
Isoelectric Point | 10.54 |
Hydrophobicity | NA |
Melting point | NA |
Half-life | between 18.9 to 28.7 min. |
Description | Recombinant human glucocerebrosidase (a lysosomal enzyme) . Elelyso used in patients with type 1 Gaucher's disease |
Indication/Disease | For the treatment of adult Type 1 Gaucher disease. |
Pharmacodynamics | Patient'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 Action | Taliglucerase 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. |
Toxicity | The most common toxic reaction seen was infusion reactions such as urticaria, arthralgia, headache, and chest pain due to IV administration. |
Metabolism | Metabolism was not determined. |
Absorption | Taliglucerase alfa is administered IV so absorption is 100%. |
| The steady state volume of distribution is between 7.30 to 11.7 L. |
Clearance | The systemic clearance was approximately 30 L/hr and 20 L/hr for 30 and 60 units/kg, respectively. |
Categories | Enzymes |
Patents Number | NA |
Date of Issue | NA |
Date of Expiry | NA |
Drug Interaction | NA |
Target | Glucocerebroside |
Brand Name | Elelyso |
Company | Pfizer |
Brand Description | Pfizer |
Prescribed For | long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease. |
Chemical Name | NA |
Formulation | The 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 Administration | Intravenous infusion |
Recommended Dosage | 60 units per kg of body weight |
Contraindication | NA |
Side Effects | pruritus, flushing, headache, arthralgia, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea, and rash. |
Useful Link 1 | Link |
Useful Link 2 | NA |
Remarks | NA |