Alglucerase

Identification

Name
Alglucerase
Accession Number
DB00088  (BTD00037, BIOD00037)
Type
Biotech
Groups
Approved, Investigational
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
Db00088
Protein chemical formula
C2532H3854N672O711S16
Protein average weight
55597.4 Da
Sequences
>DB00088 sequence
ARPCIPKSFGYSSVVCVCNATYCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANH
TGTGLLLTLQPEQKFQKVKGFGGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIR
VPMASCDFSIRTYTYADTPDDFQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWT
SPTWLKTNGAVNGKGSLKGQPGDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGL
LSGYPFQCLGFTPEHQRDFIARDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPE
AAKYVHGIAVHWYLDFLAPAKATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRG
MQYSHSIITNLLYHVVGWTDWNLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHL
GHFSKFIPEGSQRVGLVASQKNDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFL
ETISPGYSIHTYLWRRQ
Download FASTA Format
Synonyms
Not Available
International/Other Brands
Ceredase (Genzyme)
Categories
UNII
27T56C7KK0
CAS number
143003-46-7

Pharmacology

Indication

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

Structured Indications
Not Available
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 alglucerase into Gaucher disease patients leads to elevated serum levels of the enzyme and reduction in the accumulation of glucocerebroside

Mechanism of action

Alglucerase 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
  • 49.4 to 282.1 mL/kg
Protein binding
Not Available
Metabolism
Not Available
Route of elimination
Not Available
Half life

3.6-10.4 min

Clearance
Not Available
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. Grabowski GA, Kacena K, Cole JA, Hollak CE, Zhang L, Yee J, Mistry PK, Zimran A, Charrow J, vom Dahl S: Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. Genet Med. 2009 Feb;11(2):92-100. doi: 10.1097/GIM.0b013e31818e2c19. [PubMed:19265748]
External Links
UniProt
P04062
Genbank
M16328
PubChem Substance
46507500
ChEMBL
CHEMBL1201633
PharmGKB
PA164748093
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Alglucerase
ATC Codes
A16AB01 — Alglucerase
FDA label
Download (54.4 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
2CompletedTreatmentGaucher's Disease1
3CompletedTreatmentGaucher's Disease5
4CompletedTreatmentGaucher's Disease1
Not AvailableNo Longer AvailableNot AvailableGaucher's Disease1

Pharmacoeconomics

Manufacturers
  • Genzyme corp
Packagers
Dosage forms
Not Available
Prices
Unit descriptionCostUnit
Ceredase 80 unit/ml vial380.64USD ml
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Not Available

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 February 03, 2018 13:56