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
Db00090
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

Pharmacology
Reduce drug development failure rates
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Associated Conditions
Contraindications & Blackbox Warnings
Contraindications
Avoid life-threatening adverse drug events
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Avoid life-threatening adverse drug events & improve clinical decision support.
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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.

TargetActionsOrganism
AIduronic acid
other/unknown
Humans
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
Adverseeffects
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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
No interactions found.

Products

Products2
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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
AldurazymeInjection, solution, concentrate100 U/mlIntravenousGenzyme Europe Bv2016-09-08Not applicableEU flag
AldurazymeSolution0.58 mg / mLIntravenousSanofi Genzyme, a Division of Sanofi Aventis Canada Inc2004-08-12Not applicableCanada flag
AldurazymeInjection, solution, concentrate100 U/mlIntravenousGenzyme Europe Bv2016-09-08Not applicableEU flag
AldurazymeInjection, solution, concentrate2.9 mg/5mLIntravenousGenzyme Corporation2003-04-30Not applicableUS flag
AldurazymeInjection, solution, concentrate100 U/mlIntravenousGenzyme Europe Bv2016-09-08Not applicableEU flag

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
  1. 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]
  2. 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]
  3. FDA Approved Drug Products: ALDURAZYME (laronidase) injection [Link]
UniProt
P35475
Genbank
M74715
PubChem Substance
46505612
RxNav
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
PhaseStatusPurposeConditionsCount
4CompletedTreatmentHurler-Scheie Syndrome / Hurlers Syndrome / Mucopolysaccharidosis V / Type 1 Mucopolysaccharidosis2
4RecruitingTreatmentHurler-Scheie Syndrome / Hurlers Syndrome / Scheie / Type 1 Mucopolysaccharidosis1
4RecruitingTreatmentType 1 Mucopolysaccharidosis1
3CompletedTreatmentHurler-Scheie Syndrome / Hurlers Syndrome / Mucopolysaccharidosis V / Type 1 Mucopolysaccharidosis2
3CompletedTreatmentHurler-Scheie Syndrome / Hurlers Syndrome / Type 1 Mucopolysaccharidosis1
2CompletedTreatmentHurler-Scheie Syndrome / Hurlers Syndrome / Mucopolysaccharidosis V / Type 1 Mucopolysaccharidosis1
2CompletedTreatmentHurlers Syndrome / Type 1 Mucopolysaccharidosis1
1CompletedTreatmentHurlers Syndrome2
1RecruitingTreatmentGaucher Disease, Type 3 / MPS II / MPS IVA / MPS VI / MPS VII / Myocardial Perfusion Imaging / Pompe Disease (Infantile-Onset) / Type 2 Gaucher Disease / Wolman's Disease1
1TerminatedTreatmentHurler-Scheie Syndrome / Lysosomal Storage Diseases / Mucopolysaccharidosis V / Spinal Cord Compression / Type 1 Mucopolysaccharidosis1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
  • BioMarin Pharmaceuticals Inc.
  • Genzyme Inc.
  • Hollister-Stier Laboratories LLC
Dosage Forms
FormRouteStrength
Injection, solutionIntravenous2.9 mg
Injection, solution, concentrateIntravenous100 U/ml
Injection, solution, concentrateIntravenous2.9 mg/5mL
Injection, solution, concentrateIntravenous; Parenteral500 U/5ml
SolutionIntravenous0.58 mg / mL
SolutionIntravenous
SolutionIntravenous2.9 mg
Injection, solution, concentrateIntravenous
Injection, solutionIntravenous500 u/5ml
Prices
Unit descriptionCostUnit
Aldurazyme 2.9 mg/5 ml vial168.0USD ml
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Not Available

Properties

State
Liquid
Experimental Properties
PropertyValueSource
hydrophobicity-0.300Not Available
isoelectric point9.09Not Available

Targets

Drugtargets2
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Kind
Small molecule
Organism
Humans
Pharmacological action
Yes
Actions
Other/unknown
References
  1. Overington JP, Al-Lazikani B, Hopkins AL: How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. [Article]
  2. 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]
  3. 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]
  4. 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]
  5. 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 January 16, 2022 18:54