Alglucerase
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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- Approved, Investigational, Withdrawn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.
Target Actions Organism AGlucocerebroside other/unknownHumans - 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
- Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!Improve decision support & research outcomes with our structured adverse effects data.
- 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
- Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Ceredase Injection, solution, concentrate 80 U/1mL Intravenous Genzyme Corporation 1991-04-05 2011-04-01 US
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
- 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]
- 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]
- FDA Approved Drug Products: Ceredase (alglucerase) for injection [Link]
- External Links
- UniProt
- P04062
- Genbank
- M16328
- PubChem Substance
- 46507500
- 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
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample data2 Completed Treatment Gaucher Disease 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Genzyme corp
- Packagers
- Cardinal Health
- Genzyme Inc.
- Oso Biopharmaceuticals Manufacturing LLC
- Dosage Forms
Form Route Strength Injection, solution, concentrate Intravenous 80 U/1mL Solution Intravenous 400 UI Solution Intravenous 50 UI - Prices
Unit description Cost Unit Ceredase 80 unit/ml vial 380.64USD ml DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.- Patents
- Not Available
Properties
- State
- Liquid
- Experimental Properties
Property Value Source hydrophobicity -0.168 Not Available isoelectric point 7.41 Not Available
Targets
References
- Pastores GM, Hughes DA: Gaucher Disease . [Article]
Drug created at June 13, 2005 13:24 / Updated at August 02, 2024 07:21