Alglucerase

Identification

Summary

Alglucerase is a form of recombinant beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease.

Generic Name
Alglucerase
DrugBank Accession Number
DB00088
Background

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. Alglucerase was first approved by the FDA in 1991;2 however, it was later discontinued from the market.

Type
Biotech
Groups
Approved, Investigational, Withdrawn
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
Protein Structure
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
  • Alglucerase

Pharmacology

Indication

Alglucerase is indicated for use as a long-term enzyme replacement therapy in patients with Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in moderate-to-severe anemia, thrombocytopenia, bone disease, or significant hepato- or splenomegaly.3

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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
Humans
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

Adverse Effects
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Toxicity

Not Available

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
Not Available

Products

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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
CeredaseInjection, solution, concentrate80 U/1mLIntravenousGenzyme Corporation1991-04-052011-04-01US flag

Categories

ATC Codes
A16AB01 — Alglucerase
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
27T56C7KK0
CAS number
143003-46-7

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. [Article]
  2. Deegan PB, Cox TM: Imiglucerase in the treatment of Gaucher disease: a history and perspective. Drug Des Devel Ther. 2012;6:81-106. doi: 10.2147/DDDT.S14395. Epub 2012 Apr 18. [Article]
  3. FDA Approved Drug Products: Ceredase (alglucerase) for injection [Link]
UniProt
P04062
Genbank
M16328
PubChem Substance
46507500
RxNav
46049
ChEMBL
CHEMBL1201633
PharmGKB
PA164748093
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Alglucerase
FDA label
Download (54.4 KB)

Clinical Trials

Clinical Trials
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Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package
PhaseStatusPurposeConditionsCountStart DateWhy Stopped100+ additional columns
2CompletedTreatmentGaucher Disease1somestatusstop reasonjust information to hide

Pharmacoeconomics

Manufacturers
  • Genzyme corp
Packagers
  • Cardinal Health
  • Genzyme Inc.
  • Oso Biopharmaceuticals Manufacturing LLC
Dosage Forms
FormRouteStrength
Injection, solution, concentrateIntravenous80 U/1mL
SolutionIntravenous400 UI
SolutionIntravenous50 UI
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

Drug created at June 13, 2005 13:24 / Updated at August 02, 2024 07:21