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TALIGLUCERASE ALFA DB08876 C2580H3918N680O727S g/mol

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Presentation on theme: "TALIGLUCERASE ALFA DB08876 C2580H3918N680O727S g/mol"— Presentation transcript:

1 TALIGLUCERASE ALFA DB08876 C2580H3918N680O727S17 56637.9397 g/mol
CATEGORY Enzymes

2 DESCRIPTION Taliglucerase alfa is the recombinant active form of the human lysosomal enzyme, β-glucocerebrosidase. It was approved in 2012 and is marketed under the name Elelyso for use in patients with type 1 Gaucher's disease. INDICATION 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.

3 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%.

4 HALF-LIFE The half life is between 18.9 to 28.7 min. ROUTE OF ELIMINATION MMAE is eliminated by the feces (with 72% unchanged) and urine. 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. TARGETS Glucocerebroside

5 Elelyso (Previous name: Uplyso)
IV infusion 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 ) 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.

6 DOSAGE 60 units/kg via IV infusion (administered over 60 to 120 minutes) every 2 weeks FORMULATION ELELYSO is supplied as a sterile, non-pyrogenic, lyophilized product. 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. ADVERSE REACTION: Hives; difficult breathing; blue lips or fingernails; feeling like you might pass out; swelling of your face, lips, tongue, or throat

7 REFERENCES


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