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Identification
- Summary
Idursulfase is a purified lysosomal enzyme used as a replacement therapy for Hunter syndrome in pediatric and adult patients.
- Brand Names
- Elaprase
- Generic Name
- Idursulfase
- DrugBank Accession Number
- DB01271
- Background
Idursulfase is a purified form of human iduronate-2-sulfatase, a lysosomal enzyme. Idursulfase is produced by recombinant DNA technology in a human cell line. Idursulfase is an enzyme that hydrolyzes the 2-sulfate esters of terminal iduronate sulfate residues from the glycosaminoglycans dermatan sulfate and heparan sulfate in the lysosomes of various cell types. Idursulfase is a 525-amino acid glycoprotein with a molecular weight of approximately 76 kilodaltons. The enzyme contains eight asparagine-linked glycosylation sites occupied by complex oligosaccharide structures. The enzyme activity of idursulfase is dependent on the post-translational modification of a specific cysteine to formylglycine.
- Type
- Biotech
- Groups
- Approved
- Biologic Classification
- Protein Based Therapies
Recombinant Enzymes - Protein Chemical Formula
- C2654H4000N688O774S14
- Protein Average Weight
- 76000.0 Da
- Sequences
>Idursulfase heavy chain SETQANSTTDALNVLLIIVDDLRPSLGCYGDKLVRSPNIDQLASHSLLFQNAFAQQAVCA PSRVSFLTGRRPDTTRLYDFNSYWRVHAGNFSTIPQYFKENGYVTMSVGKVFHPGISSNH TDDSPYSWSFPPYHPSSEKYENTKTCRGPDGELHANLLCPVDVLDVPEGTLPDKQSTEQA IQLLEKMKTSASPFFLAVGYHKPHIPFRYPKEFQKLYPLENITLAPDPEVPDGLPPVAYN PWMDIRQREDVQALNISVPYGPIPVDFQRKIRQSYFASVSYLDTQVGRLLSALDDLQLAN STIIAFTSDHGWALGEHGEWAKYSNFDVATHVPLIFYVPGRTASLPEAGEKLFPYLDPFD SASQLMEPGRQSMDLVELVSLFPTLAGLAGLQVPPRCPVPSFHVELCREGKNLLKHFRFR DLEEDPYLPG
>Idursulfase light chain NPRELIAYSQYPRPSDIPQWNSDKPSLKDIKIMGYSIRTIDYRYTVWVGFNPDEFLANFS DIHAGELYFVDSDPLQDHNMYNDSQGGDLFQLLMP
Download FASTA Format- Synonyms
- Alpha-L-iduronate sulfate sulfatase
- Iduronate 2-sulfatase
- Idursulfasa
- Idursulfase
Pharmacology
- Indication
For the treatment of Hunter syndrome in adults and children ages 5 and older.
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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Management of Mucopolysaccharidosis ii •••••••••••• - Contraindications & Blackbox Warnings
Prevent Adverse Drug Events TodayTap into our Clinical API for life-saving information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.Avoid life-threatening adverse drug events with our Clinical API- Pharmacodynamics
Idursulfase is a purified form of the lysosomal enzyme human iduronate-2-sulfatase of recombinant DNA origin. It is designed to replace the natural enzyme, increasing catabolism of certain accumulated glycosaminoglycans (GAG), which abnormally accumulate in multiple tissue types in patients with mucopolysaccharidosis II (MPS-II, or Hunter syndrome).
- Mechanism of action
Hunter's Syndrome is an X-linked recessive disease caused by insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. This enzyme cleaves the terminal 2-O-sulfate moieties from the glycosaminoglycans (GAG) dermatan sulfate and heparan sulfate. Due to the missing or defective iduronate-2-sulfatase enzyme in patients with Hunter's Syndrome, GAG progressively accumulate in the lysosomes of a variety of cells, leading to cellular engorgement, organomegaly, tissue destruction and organ system dysfunction. Treatment of Hunter's Syndrome patients with idursulfase provides exogenous enzyme for uptake into cellular lysosomes. Targeting of idursulfase to the lysosome occurs by endocytosis from the cell surface. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow specific binding of the enzymes to the M6P receptors on the cell surface, leading to cellular internalization of the enzyme, targeting to intracellular lysosomes and subsequent catabolism of accumulated GAG.
Target Actions Organism ADermatan sulfate Not Available Humans AHeparan sulfate Not Available Humans NPerilipin-3 Not Available Humans - Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
44 ± 19 minutes
- Clearance
- 3 mL/min/kg [Patients (7.7 – 27 years) with Hunter syndrome with treatment week 1(0.5 mg/kg ELAPRASE administered weekly as a 3-hour infusion)]
- 3.4 mL/min/kg [patients (7.7 – 27 years) with Hunter syndrome with treatment week 27 (0.5 mg/kg ELAPRASE administered weekly as a 3-hour infusion)]
- 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
There is no experience with overdosage of Idursulfase in humans. Single intravenous doses of idursulfase up to 20 mg/kg were not lethal in male rats and cynomolgus monkeys (approximately 6.5 and 13 times, respectively, of the recommended human dose based on body surface area) and there were no clinical signs of toxicity.
- 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
- No interactions found.
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 Elaprase Injection, solution, concentrate 2 mg/ml Intravenous Takeda Pharmaceuticals International Ag Ireland Branch 2021-02-11 Not applicable EU 
Elaprase Solution 2 mg / mL Intravenous Takeda 2007-08-01 Not applicable Canada 
Elaprase Injection, solution, concentrate 2 mg/ml Intravenous Takeda Pharmaceuticals International Ag Ireland Branch 2021-02-11 Not applicable EU Elaprase Solution, concentrate 6 mg/3mL Intravenous Takeda Pharmaceuticals America, Inc. 2006-07-24 Not applicable US 
Elaprase Injection, solution, concentrate 2 mg/ml Intravenous Takeda Pharmaceuticals International Ag Ireland Branch 2021-02-11 Not applicable EU
Categories
- ATC Codes
- A16AB09 — Idursulfase
- 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
- 5W8JGG2651
- CAS number
- 50936-59-9
References
- General References
- Garcia AR, DaCosta JM, Pan J, Muenzer J, Lamsa JC: Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II. Mol Genet Metab. 2007 Jun;91(2):183-90. Epub 2007 Apr 24. [Article]
- Zareba G: Idursulfase in Hunter syndrome treatment. Drugs Today (Barc). 2007 Nov;43(11):759-67. doi: 10.1358/dot.2007.43.11.1157619. [Article]
- Clarke LA: Idursulfase for the treatment of mucopolysaccharidosis II. Expert Opin Pharmacother. 2008 Feb;9(2):311-7. doi: 10.1517/14656566.9.2.311 . [Article]
- Burrow TA, Leslie ND: Review of the use of idursulfase in the treatment of mucopolysaccharidosis II. Biologics. 2008 Jun;2(2):311-20. [Article]
- Scarpa M: Mucopolysaccharidosis Type II . [Article]
- Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, Meldgaard Lund A, Malm G, Van der Ploeg AT, Zeman J: Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008 Mar;167(3):267-77. Epub 2007 Nov 23. [Article]
- External Links
- PubChem Substance
- 46507347
- 644101
- ChEMBL
- CHEMBL1201826
- PharmGKB
- PA164749134
- Drugs.com
- Drugs.com Drug Page
- Wikipedia
- Idursulfase
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 data4 Active Not Recruiting Treatment Mucopolysaccharidosis Type II (MPS II) 2 somestatus stop reason just information to hide 4 Completed Treatment Mucopolysaccharidosis Type II (MPS II) 1 somestatus stop reason just information to hide 4 Recruiting Treatment Mucopolysaccharidosis (MPS) / Mucopolysaccharidosis Type II (MPS II) 1 somestatus stop reason just information to hide 3 Recruiting Treatment Mucopolysaccharidosis Type II (MPS II) 1 somestatus stop reason just information to hide 2 Unknown Status Treatment Mucopolysaccharidosis Type II (MPS II) 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Baxter International Inc.
- Shire Inc.
- Dosage Forms
Form Route Strength Injection Intravenous Injection, solution, concentrate Intravenous 2 MG/ML Injection, solution, concentrate Intravenous; Parenteral 2 MG/ML Solution Intravenous 2 mg / mL Solution, concentrate Intravenous 6 mg/3mL Solution Intravenous 6.0 mg Solution Intravenous 6 mg Injection, solution Intravenous Solution Intravenous 6 mg/1vial - Prices
Unit description Cost Unit Elaprase 6 mg/3 ml vial 1051.28USD ml DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.- Patents
- Not Available
Properties
- State
- Solid
- Experimental Properties
- Not Available
Targets
References
- Burrow TA, Leslie ND: Review of the use of idursulfase in the treatment of mucopolysaccharidosis II. Biologics. 2008 Jun;2(2):311-20. [Article]
- Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, Meldgaard Lund A, Malm G, Van der Ploeg AT, Zeman J: Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008 Mar;167(3):267-77. Epub 2007 Nov 23. [Article]
- Garcia AR, Pan J, Lamsa JC, Muenzer J: The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J Inherit Metab Dis. 2007 Nov;30(6):924-34. Epub 2007 Sep 16. [Article]
References
- Burrow TA, Leslie ND: Review of the use of idursulfase in the treatment of mucopolysaccharidosis II. Biologics. 2008 Jun;2(2):311-20. [Article]
- Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, Meldgaard Lund A, Malm G, Van der Ploeg AT, Zeman J: Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008 Mar;167(3):267-77. Epub 2007 Nov 23. [Article]
- Garcia AR, Pan J, Lamsa JC, Muenzer J: The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J Inherit Metab Dis. 2007 Nov;30(6):924-34. Epub 2007 Sep 16. [Article]
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- No
- General Function
- Structural component of lipid droplets, which is required for the formation and maintenance of lipid storage droplets (PubMed:34077757). Required for the transport of mannose 6-phosphate receptors (MPR) from endosomes to the trans-Golgi network (PubMed:9590177)
- Specific Function
- Cadherin binding
- Gene Name
- PLIN3
- Uniprot ID
- O60664
- Uniprot Name
- Perilipin-3
- Molecular Weight
- 47074.665 Da
References
- Overington JP, Al-Lazikani B, Hopkins AL: How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. [Article]
- Imming P, Sinning C, Meyer A: Drugs, their targets and the nature and number of drug targets. Nat Rev Drug Discov. 2006 Oct;5(10):821-34. [Article]
- Clarke LA: Idursulfase for the treatment of mucopolysaccharidosis II. Expert Opin Pharmacother. 2008 Feb;9(2):311-7. doi: 10.1517/14656566.9.2.311 . [Article]
- Burrow TA, Leslie ND: Review of the use of idursulfase in the treatment of mucopolysaccharidosis II. Biologics. 2008 Jun;2(2):311-20. [Article]
- Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, Meldgaard Lund A, Malm G, Van der Ploeg AT, Zeman J: Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008 Mar;167(3):267-77. Epub 2007 Nov 23. [Article]
Drug created at May 16, 2007 20:17 / Updated at June 03, 2022 07:24