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.

Pharmacology
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Associated Conditions
Contraindications & Blackbox Warnings
Contraindications
Avoid life-threatening adverse drug events
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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.

TargetActionsOrganism
UGlucocerebrosideNot AvailableHumans
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
Adverseeffects
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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

Products2
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International/Other Brands
Uplyso (PFIZER)
Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
ElelysoPowder, for solution200 unit / vialIntravenousPfizer Canada Ulc2014-09-08Not applicableCanada flag
ElelysoInjection, powder, lyophilized, for solution200 U/5mLIntravenousPfizer Laboratories Div Pfizer Inc2012-05-01Not applicableUS flag

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
  1. 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]
  2. 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]
KEGG Drug
D09675
PubChem Substance
347910377
RxNav
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
PhaseStatusPurposeConditionsCount
4CompletedTreatmentGaucher's Disease1
4RecruitingTreatmentGaucher Disease, Type 31
4WithdrawnOtherGaucher Disease, Type 11
3CompletedTreatmentGaucher's Disease5
2CompletedTreatmentGaucher's Disease1
1CompletedTreatmentGaucher's Disease2
Not AvailableActive Not RecruitingTreatmentGaucher Disease, Type 11
Not AvailableNo Longer AvailableNot AvailableGaucher's Disease1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
Injection, powder, lyophilized, for solutionIntravenous200 U/5mL
Powder, for solutionIntravenous200 unit / vial
Injection
Prices
Not Available
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)Region
US8790641No2014-07-292025-10-18US flag
US8741620No2014-06-032024-02-24US flag
US8227230No2012-07-242024-02-24US flag

Properties

State
Solid
Experimental Properties
PropertyValueSource
water solubilitySoluble in water sodium chloride solutions and ethanol.From MSDS.

Targets

Drugtargets2
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1. Glucocerebroside
Kind
Small molecule
Organism
Humans
Pharmacological action
Unknown
References
  1. 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]
  2. 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 January 19, 2022 01:13