Imiglucerase

Identification

Name
Imiglucerase
Accession Number
DB00053  (BTD00026, BIOD00026)
Type
Biotech
Groups
Approved
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
Description

Human Beta-glucocerebrosidase or Beta-D-glucosyl-N-acylsphingosine glucohydrolase E.C. 3.2.1.45. 497 residue protein with N-linked carbohydrates, MW=59.3 kD. Alglucerase is prepared by modification of the oligosaccharide chains of human Beta-glucocerebrosidase. The modification alters the sugar residues at the non-reducing ends of the oligosaccharide chains of the glycoprotein so that they are predominantly terminated with mannose residues.

Protein structure
Db00053
Protein chemical formula
C2532H3854N672O711S16
Protein average weight
55597.4 Da
Sequences
>DB00053 sequence
ARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANH
TGTGLLLTLQPEQKFQKVKGFGGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIR
VPMASCDFSIRTYTYADTPDDFQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWT
SPTWLKTNGAVNGKGSLKGQPGDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGL
LSGYPFQCLGFTPEHQRDFIARDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPE
AAKYVHGIAVHWYLDFLAPAKATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRG
MQYSHSIITNLLYHVVGWTDWNLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHL
GHFSKFIPEGSQRVGLVASQKNDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFL
ETISPGYSIHTYLWRRQ
Download FASTA Format
Synonyms
  • Imiglucerasa
External IDs
ISU-302
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
CerezymeInjection, powder, lyophilized, for solution40 U/1mLIntravenousGenzyme Corporation1994-05-23Not applicableUs
CerezymePowder, for solution400 unitIntravenousSanofi Genzyme, a Division of Sanofi Aventis Canada Inc2000-06-14Not applicableCanada
CerezymeInjection, powder, lyophilized, for solution40 U/1mLIntravenousGenzyme Corporation1994-05-23Not applicableUs
CerezymePowder, for solution200 unitIntravenousSanofi Genzyme, a Division of Sanofi Aventis Canada Inc1997-05-012016-08-04Canada
Categories
UNII
Q6U6J48BWY
CAS number
154248-97-2

Pharmacology

Indication

For the treatment of Gaucher's disease (deficiency in glucocerebrosidase)

Associated Conditions
Pharmacodynamics

Gaucher disease is characterized by a functional deficiency in Beta-glucocerebrosidase enzymatic activity and the resultant accumulation of lipid glucocerebroside in tissue macrophages which become engorged and are termed Gaucher cells. Gaucher cells are typically found in liver, spleen and bone marrow. This can lead to an enlarged spleen and liver (hepatosplenomegaly). Secondary hematologic sequelae include severe anemia and thrombocytopenia. Injections of imiglucerase into Gaucher disease patients leads to elevated serum levels of the enzyme and reduction in the accumulation of glucocerebroside leading to reduced anemia and thrombocytopenia, reduced spleen and liver size, and decreased cachexia

Mechanism of action

Imiglucerase catalyzes the hydrolysis of the glycolipid, glucocerebroside, to glucose and ceramide as part of the normal degradation pathway for membrane lipids.

TargetActionsOrganism
AGlucocerebroside
other/unknown
Human
Absorption
Not Available
Volume of distribution
  • 0.09 to 0.15 L/kg
Protein binding
Not Available
Metabolism
Not Available
Route of elimination
Not Available
Half life

3.6-10.4 min

Clearance
  • 14.5 +/- 4.0 mL/min/kg
Toxicity
Not Available
Affected organisms
  • Humans and other mammals
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
No interactions found.
Food Interactions
Not Available

References

General References
  1. Pastores GM, Hughes DA: Gaucher Disease . [PubMed:20301446]
External Links
UniProt
P04062
Genbank
M16328
KEGG Drug
D02810
PubChem Substance
46508744
ChEMBL
CHEMBL1201632
PharmGKB
PA164742934
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Imiglucerase
ATC Codes
A16AB02 — Imiglucerase
AHFS Codes
  • 44:00.00 — Enzymes
FDA label
Download (111 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
2Unknown StatusTreatmentGaucher's Disease1
3CompletedTreatmentGaucher Disease, Type 13
3RecruitingTreatmentGaucher's Disease Type 1 / Gaucher's disease type III1
3WithdrawnNot AvailableGaucher's Disease1
4CompletedTreatmentCerebroside Lipidosis Syndrome / Clucocerebrosidase Deficiency Disease / Gaucher Disease, Non-Neuronopathic Form / Gaucher's Disease Type 1 / Glucosylceramide Beta-Glucosidase Deficiency Disease1
4CompletedTreatmentCerebroside Lipidosis Syndrome / Gaucher Disease, Non-Neuronopathic Form / Gaucher Disease, Type 1 / Glucocerebrosidase Deficiency Disease / Glucosylceramide Beta-Glucosidase Deficiency Disease1
Not AvailableCompletedNot AvailableGaucher's Disease1

Pharmacoeconomics

Manufacturers
  • Genzyme corp
Packagers
  • Genzyme Inc.
Dosage forms
FormRouteStrength
Injection, powder, lyophilized, for solutionIntravenous40 U/1mL
Powder, for solutionIntravenous200 unit
Powder, for solutionIntravenous400 unit
Prices
Unit descriptionCostUnit
Cerezyme 400 unit vial1903.2USD vial
Cerezyme 200 unit vial951.6USD vial
Ceredase 80 unit/ml vial380.64USD ml
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)
US5549892No1993-08-272013-08-27Us

Properties

State
Liquid
Experimental Properties
PropertyValueSource
hydrophobicity-0.168Not Available
isoelectric point7.41Not Available

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
Yes
Actions
Other/unknown
References
  1. Pastores GM, Hughes DA: Gaucher Disease . [PubMed:20301446]

Drug created on June 13, 2005 07:24 / Updated on November 13, 2018 07:44