A database of FDA approved therapeutic peptides and proteins
Details of Th1131 which contains 1 entries. |
Entry 1 | |
(1) Primary information | |
---|---|
ID | 1608 |
ThPP ID | Th1131 |
Therapeutic Peptide/Protein Name | Taliglucerase alfa |
Sequence | EFARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRR view full sequnce in fasta |
Functional Classification | Ia |
Molecular Weight | 56637.9397 |
Chemical Formula | C2580H3918N680O727S17 |
Isoelectric Point | 10.54 |
Hydrophobicity | N.A. |
Melting Point (℃) | N.A. |
Half Life | N.A. |
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%. |
Volume of Distribution | 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 | N.A. |
Date of Issue | N.A. |
Date of Expiry | N.A. |
Drug Interaction | N.A. |
Target | Glucocerebroside |
Information of corresponding available drug in the market | |
Brand Name | Elelyso |
Company | Pfizer |
Brand Discription | Taliglucerase 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 for | long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease. |
Chemical Name | N.A. |
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. |
Physcial 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 | N.A. |
Side Effects | pruritus, flushing, headache, arthralgia, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea, and rash. |
Useful Link | http://www.rxlist.com/elelyso-drug.htm http://www.elelyso.com/ http://www.drugs.com/elelyso.html |
PubMed ID | 23430813, 25453586, 24950666, 24630271, 23199589, 21900191 |
3-D Structure | Th1131 (View) or (Download) |