Taliglucerase alfa

Identification

Name
Taliglucerase alfa
Accession Number
DB08876  (DB06667)
Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
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.

Protein chemical formula
C2580H3918N680O727S17
Protein average weight
56637.9397 Da
Sequences
Not Available
Synonyms
  • Glucosylcerebrosidase
  • prGC-D
  • prGCD
External IDs
PRX-112
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
ElelysoPowder, for solution200 unitIntravenousPfizer2014-09-08Not applicableCanada
ElelysoInjection, powder, lyophilized, for solution200 U/5mLIntravenousPfizer Laboratories Div Pfizer Inc.2012-05-01Not applicableUs
International/Other Brands
Uplyso (PFIZER)
Categories
UNII
0R4NLX88O4
CAS number
37228-64-1

Pharmacology

Indication

For the treatment of adult Type 1 Gaucher disease.

Associated Conditions
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 AvailableHuman
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.

Toxicity

The most common toxic reaction seen was infusion reactions such as urticaria, arthralgia, headache, and chest pain due to IV administration.

Affected organisms
  • Humans and other mammals
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
No interactions found.
Food Interactions
  • No food effects found.

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. [PubMed:21900191]
  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. [PubMed:19277123]
External Links
KEGG Drug
D09675
PubChem Substance
347910377
ChEMBL
CHEMBL1964120
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Taliglucerase_alfa
ATC Codes
A16AB11 — Taliglucerase alfa
AHFS Codes
  • 44:00.00 — Enzymes
FDA label
Download (77.6 KB)
MSDS
Download (28.3 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1CompletedTreatmentGaucher's Disease2
2CompletedTreatmentGaucher's Disease1
2Unknown StatusTreatmentGaucher's Disease1
3CompletedTreatmentGaucher's Disease5
4CompletedTreatmentGaucher's Disease1
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
Prices
Not Available
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)
US8790641No2005-10-182025-10-18Us
US8741620No2004-02-242024-02-24Us
US8227230No2004-02-242024-02-24Us

Properties

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

Taxonomy

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

Targets

1. Glucocerebroside
Kind
Small molecule
Organism
Human
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. [PubMed:22916340]
  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. [PubMed:17524049]

Drug created on May 12, 2013 21:59 / Updated on October 16, 2018 08:40