Laronidase
Identification
- Summary
Laronidase is a form of recombinant human alpha-L-iduronidase used to treat Hurler and Hurler-Scheie forms of mucopolysaccharidosis, a genetic deficiency of alpha-L-iduronidase.
- Brand Names
- Aldurazyme
- Generic Name
- Laronidase
- DrugBank Accession Number
- DB00090
- Background
Human recombinant alpha-L-iduronidase, 628 residues (mature form), produced by recombinant DNAtechnology in a Chinese hamster ovary cell line. Laronidase is a glycoprotein with a molecular weight of approximately 83 kD. The predicted amino acid sequence of the recombinant form, as well as the nucleotide sequence that encodes it, are identical to a polymorphic form of human a-L-iduronidase. It contains 6 N-linked oligosaccharide modification sites.
- Type
- Biotech
- Groups
- Approved
- Biologic Classification
- Protein Based Therapies
Recombinant Enzymes - Protein Structure
- Protein Chemical Formula
- C3160H4848N898O881S12
- Protein Average Weight
- 69899.4 Da
- Sequences
>DB00090 sequence APHLVQVDAARALWPLRRFWRSTGFCPPLPHSQADQYVLSWDQQLNLAYVGAVPHRGIKQ VRTHWLLELVTTRGSTGRGLSYNFTHLDGYLDLLRENQLLPGFELMGSASGHFTDFEDKQ QVFEWKDLVSSLARRYIGRYGLAHVSKWNFETWNEPDHHDFDNVSMTMQGFLNYYDACSE GLRAASPALRLGGPGDSFHTPPRSPLSWGLLRHCHDGTNFFTGEAGVRLDYISLHRKGAR SSISILEQEKVVAQQIRQLFPKFADTPIYNDEADPLVGWSLPQPWRADVTYAAMVVKVIA QHQNLLLANTTSAFPYALLSNDNAFLSYHPHPFAQRTLTARFQVNNTRPPHVQLLRKPVL TAMGLLALLDEEQLWAEVSQAGTVLDSNHTVGVLASAHRPQGPADAWRAAVLIYASDDTR AHPNRSVAVTLRLRGVPPGPGLVYVTRYLDNGLCSPDGEWRRLGRPVFPTAEQFRRMRAA EDPVAAAPRPLPAGGRLTLRPALRLPSLLLVHVCARPEKPPGQVTRLRALPLTQGQLVLV WSDEHVGSKCLWTYEIQFSQDGKAYTPVSRKPSTFNLFVFSPDTGAVSGSYRVRALDYWA RPGPFSDPVPYLEVPVPRGPPSPGNP
Download FASTA Format- Synonyms
- alpha-L-Idosiduronase
- Human Recombinant alpha-L-iduronidase
- Laronidasa
- Laronidase
- Laronidase (genetical recombination)
Pharmacology
- Indication
For the treatment of mucopolysaccharidosis
Reduce drug development failure ratesBuild, train, & validate machine-learning modelswith evidence-based and structured datasets.Build, train, & validate predictive machine-learning models with structured datasets.- Associated Conditions
Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Management of Type 1 mucopolysaccharidosis •••••••••••• •••••• ••••••••• •••••••• •• •••••• •••••••• •••••••••• •••••••• Management of Type 1 mucopolysaccharidosis •••••••••••• •••••• ••••••••• •••••••••• •••••••• Management of Type 1 mucopolysaccharidosis •••••••••••• •••••• ••••••••• •••••••••• •••••••• - 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
Laronidase is used to treat mucopolysaccharide storage disorders (specifically mucopolysaccharidosis 1 or Hurlers syndrome) caused by deficiencies of alpha-L-iduronidase. Reduced or absent a-L-iduronidase activity results in the accumulation of the GAG substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.
- Mechanism of action
Laronidase catalyses the hydrolysis of terminal alpha-L-iduronic acid residues of dermatan sulfate and heparin sulfate.
Target Actions Organism AIduronic acid other/unknownHumans - Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
1.5-3.6 hrs
- 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 Browse all" title="About SNP Mediated Effects/ADRs" id="snp-actions-info" class="drug-info-popup" href="javascript:void(0);">
- Not Available
Interactions
- Drug Interactions Learn More" title="About Drug Interactions" id="structured-interactions-info" class="drug-info-popup" href="javascript:void(0);">
- 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 Aldurazyme Injection, solution, concentrate 100 U/ml Intravenous Sanofi B.V. 2016-09-08 Not applicable EU Aldurazyme Solution 0.58 mg / mL Intravenous Sanofi Genzyme, a Division of Sanofi Aventis Canada Inc 2004-08-12 Not applicable Canada Aldurazyme Injection, solution, concentrate 100 U/ml Intravenous Sanofi B.V. 2016-09-08 Not applicable EU Aldurazyme Injection, solution, concentrate 2.9 mg/5mL Intravenous Genzyme Corporation 2003-04-30 Not applicable US Aldurazyme Injection, solution, concentrate 100 U/ml Intravenous Sanofi B.V. 2016-09-08 Not applicable EU
Categories
- ATC Codes
- A16AB05 — Laronidase
- 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
- WP58SVM6R4
- CAS number
- 210589-09-6
References
- General References
- Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Sidman M, Kakkis ED, Cox GF: Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847. [Article]
- Landry Y, Gies JP: Drugs and their molecular targets: an updated overview. Fundam Clin Pharmacol. 2008 Feb;22(1):1-18. doi: 10.1111/j.1472-8206.2007.00548.x. [Article]
- FDA Approved Drug Products: ALDURAZYME (laronidase) injection [Link]
- External Links
- UniProt
- P35475
- Genbank
- M74715
- PubChem Substance
- 46505612
- 392509
- ChEMBL
- CHEMBL1201595
- PharmGKB
- PA164776670
- RxList
- RxList Drug Page
- Drugs.com
- Drugs.com Drug Page
- Wikipedia
- Iduronidase
- FDA label
- Download (43.2 KB)
Clinical Trials
- Clinical Trials Learn More" title="About Clinical Trials" id="clinical-trials-info" class="drug-info-popup" href="javascript:void(0);">
Phase Status Purpose Conditions Count 4 Completed Treatment Hurler-Scheie Syndrome / Mucopolysaccharidosis IH / Mucopolysaccharidosis Type I (MPS I) / Scheie Syndrome 2 4 Completed Treatment Mucopolysaccharidosis Type I (MPS I) 1 4 Recruiting Treatment Hurler-Scheie Syndrome / Mucopolysaccharidosis IH / Mucopolysaccharidosis Type I (MPS I) / Scheie Syndrome 1 3 Completed Treatment Hurler-Scheie Syndrome / Mucopolysaccharidosis IH / Mucopolysaccharidosis Type I (MPS I) 1 3 Completed Treatment Hurler-Scheie Syndrome / Mucopolysaccharidosis IH / Mucopolysaccharidosis Type I (MPS I) / Scheie Syndrome 2
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- BioMarin Pharmaceuticals Inc.
- Genzyme Inc.
- Hollister-Stier Laboratories LLC
- Dosage Forms
Form Route Strength Injection, solution Intravenous 2.9 mg Injection, solution, concentrate Intravenous 100 U/ml Injection, solution, concentrate Intravenous 2.9 mg/5mL Injection, solution, concentrate Intravenous; Parenteral 500 U/5ml Solution Intravenous 0.58 mg / mL Solution Intravenous Solution Intravenous 2.9 mg Injection, solution, concentrate Intravenous 100 IU/mL Injection, solution Intravenous 500 u/5ml - Prices
Unit description Cost Unit Aldurazyme 2.9 mg/5 ml vial 168.0USD 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 -0.300 Not Available isoelectric point 9.09 Not Available
Targets
![](https://go.drugbank.com/assets/locked/DrugTargets2-682eb846d210ae4bf33ca03febccfb57bb1c61318e699b729e5df4dfab2c5b67.png)
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]
- Kloska A, Bohdanowicz J, Konopa G, Tylki-Szymnska A, Jakobkiewicz-Banecka J, Czartoryska B, Liberek A, Wegrzyn A, Wegrzyn G: Changes in hair morphology of mucopolysaccharidosis I patients treated with recombinant human alpha-L-iduronidase (laronidase, Aldurazyme). Am J Med Genet A. 2005 Dec 15;139(3):199-203. [Article]
- Bach G, Eisenberg F Jr, Cantz M, Neufeld EF: The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. Proc Natl Acad Sci U S A. 1973 Jul;70(7):2134-8. [Article]
- Freeman C, Hopwood JJ: Human alpha-L-iduronidase. Catalytic properties and an integrated role in the lysosomal degradation of heparan sulphate. Biochem J. 1992 Mar 15;282 ( Pt 3):899-908. [Article]
Drug created at June 13, 2005 13:24 / Updated at February 20, 2024 23:55