Identification
- Summary
Taliglucerase alfa is a hydrolytic lysosomal glucocerebroside specific enzyme, used for long term enzyme replacement therapy in patients with Type 1 Gaucher disease.
- Brand Names
- Elelyso
- Generic Name
- Taliglucerase alfa
- DrugBank Accession Number
- DB08876
- Background
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.
- Type
- Biotech
- Groups
- Approved, Investigational
- Biologic Classification
- Protein Based Therapies
Recombinant Enzymes - Protein Chemical Formula
- C2580H3918N680O727S17
- Protein Average Weight
- 56637.9397 Da
- Sequences
- Not Available
- Synonyms
- Glucosylcerebrosidase
- prGC-D
- prGCD
- Taliglucerase alfa
- External IDs
- PRX-112
Pharmacology
- Indication
For the treatment of adult Type 1 Gaucher disease.
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- Contraindications & Blackbox Warnings
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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.
Target Actions Organism UGlucocerebroside Not Available Humans - 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.
- Protein binding
Plasma protein binding was not quantified.
- Metabolism
Metabolism was not determined.
- Route of elimination
Route of elimination was not determined.
- Half-life
The half life is between 18.9 to 28.7 min.
- Clearance
The systemic clearance was approximately 30 L/hr and 20 L/hr for 30 and 60 units/kg, respectively.
- Adverse Effects
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates.Improve decision support & research outcomes with our structured adverse effects data.- Toxicity
The most common toxic reaction seen was infusion reactions such as urticaria, arthralgia, headache, and chest pain due to IV administration.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.No interactions found.
- Food Interactions
- Take with or without food.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- International/Other Brands
- Uplyso (PFIZER)
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Elelyso Powder, for solution 200 unit / vial Intravenous Pfizer Canada Ulc 2014-09-08 Not applicable Canada 
Elelyso Injection, powder, lyophilized, for solution 200 U/5mL Intravenous Pfizer Laboratories Div Pfizer Inc 2012-05-01 Not applicable US 
Categories
- ATC Codes
- A16AB11 — Taliglucerase alfa
- Drug Categories
- Chemical TaxonomyProvided by Classyfire
- Description
- Not Available
- Kingdom
- Organic Compounds
- Super Class
- Organic Acids
- Class
- Carboxylic Acids and Derivatives
- Sub Class
- Amino Acids, Peptides, and Analogues
- Direct Parent
- Peptides
- Alternative Parents
- Not Available
- Substituents
- Not Available
- Molecular Framework
- Not Available
- External Descriptors
- Not Available
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- N69L6LM4QU
- CAS number
- 37228-64-1
References
- Synthesis Reference
Shaaltiel Y, Bartfeld D, Hashmueli S, Baum G, Brill-Almon E, Galili G, Dym O, Boldin-Adamsky SA, Silman I, Sussman JL, Futerman AH, Aviezer D: Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system. Plant Biotechnol J. 2007 Sep;5(5):579-90. Epub 2007 May 24.
- General References
- Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Munoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D: Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6. [Article]
- Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E: A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation. PLoS One. 2009;4(3):e4792. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11. [Article]
- External Links
- KEGG Drug
- D09675
- PubChem Substance
- 347910377
- 1291609
- ChEMBL
- CHEMBL1964120
- RxList
- RxList Drug Page
- Drugs.com
- Drugs.com Drug Page
- Wikipedia
- Taliglucerase_alfa
- FDA label
- Download (77.6 KB)
- MSDS
- Download (28.3 KB)
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 4 Active Not Recruiting Treatment Gaucher Disease, Type III 1 4 Completed Treatment Gaucher Disease 1 4 Withdrawn Other Gaucher Disease, Type 1 1 3 Completed Treatment Gaucher Disease 5 2 Completed Treatment Gaucher Disease 1 1 Completed Treatment Gaucher Disease 2 Not Available Completed Treatment Gaucher Disease, Type 1 1 Not Available No Longer Available Not Available Gaucher Disease 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Injection, powder, lyophilized, for solution Intravenous 200 U/5mL Powder, for solution Intravenous 200 unit / vial Injection 200 U - Prices
- Not Available
- Patents
Patent Number Pediatric Extension Approved Expires (estimated) Region US8790641 No 2014-07-29 2025-10-18 US US8741620 No 2014-06-03 2024-02-24 US US8227230 No 2012-07-24 2024-02-24 US
Properties
- State
- Solid
- Experimental Properties
Property Value Source water solubility Soluble in water sodium chloride solutions and ethanol. From MSDS.
Targets
References
- Haddley K: Taliglucerase alfa for the treatment of Gaucher's disease. Drugs Today (Barc). 2012 Aug;48(8):525-32. doi: 10.1358/dot.2012.48.8.1844808. [Article]
- Shaaltiel Y, Bartfeld D, Hashmueli S, Baum G, Brill-Almon E, Galili G, Dym O, Boldin-Adamsky SA, Silman I, Sussman JL, Futerman AH, Aviezer D: Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system. Plant Biotechnol J. 2007 Sep;5(5):579-90. Epub 2007 May 24. [Article]
Drug created at May 13, 2013 03:59 / Updated at June 03, 2022 07:24