Identification

Summary

Elosulfase alfa is a lysosomal glycosaminoglycan (GAG)-specific enzyme indicated as an enzyme replacement therapy for Mucopolysaccharidosis type IV A.

Brand Names
Vimizim
Generic Name
Elosulfase alfa
DrugBank Accession Number
DB09051
Background

Elosulfase alfa is a synthetic version of the enzyme N-acetylgalactosamine-6-sulfatase. It was approved by the FDA in 2014 for the treatment of Morquio syndrome. Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and is marketed under the brand Vimizim™. The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours, based on infusion volume, once every week.

Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Protein Based Therapies
Recombinant Enzymes
Protein Structure
Protein Chemical Formula
C5020H7588N1364O1418S34
Protein Average Weight
110800.0 Da
Sequences
>Elosulfase-alfa
APQPPNILLLLMDDMGWGDLGVYGEPSRETPLCSPSRAALLTGRLPIRNGFYTTNAHARN
LLKKAGYVSKIVGKWHLGHRPQFHPLKHGFNIPVYRDWEMVGRYYEEFPINLKTGEANLT
FLYWAVDATHAPVYASKPFLGTSQRGRYGDVADNTFVFFTSDNGAALISAPEQGGSNGPF
PGHVTAGQVSHQLGSIMDLFTTSLALAGLTLMDRPIFYYRGDTLMAATLGQHKAHFWTWT
VTTHNLEDHTKLPLIFHLGRDPGERFPLSFEALVPAQPQLNVCNWAVMNWAPPGCEKLGK
PNLDRMAAEGLLFPNFYSANAYTPQEIVGGIPDSEQLLPEDEWFGSPNCHFGPYDNKARP
QIYLQEALDFIKRQARHHPFAVREIDDSIGKILELLQDLHLCGKQTTFEGGMREPALAWW
PPSDRAIDGLNLLPTLLQGRNSWENFRQGIDFCPGQNVSGASAEYQEALSRITSVVQQHQ
CLTPPESIPKKCLWSH
Download FASTA Format
Synonyms
  • Chondroitin sulfatase
  • Chondroitinase
  • Chondrosulfatase
  • Elosulfase alfa
  • Recombinant human N-acetylgalactosamine-6-sulfatase
  • Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
  • rhGALNS
External IDs
  • BMN 110
  • BMN-110
  • J1322
  • rhGALNS

Pharmacology

Indication

Vimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

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Associated Conditions
Contraindications & Blackbox Warnings
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Pharmacodynamics

AUC: 238 min x μg/mL, standard deviation 100.

Mechanism of action

Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine-6-sulfatase activity. The sulfatase activity deficiency results in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body. The accumulation leads to widespread cellular, tissue, and organ dysfunction. Vimizim is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S. Elosulfase alfa uptake by cells into lysosomes is mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of elosulfase alfa to mannose-6-phosphate receptors.

In the absence of an animal disease model that recapitulates the human disease phenotype, elosulfase alfa pharmacological activity was evaluated using human primary chondrocytes from two MPS IVA patients. Treatment of MPS IVA chondrocytes with elosulfase alfa induced clearance of KS lysosomal storage from the chondrocytes.

Absorption

Cmax: 1.49 μg/mL, standard deviation 0.534.

Volume of distribution

396 mL/kg, standard deviation 316.

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

week 0: 7.52 min week 22: 35.9 min

Clearance

10.0 mL/min/kg. (standard deviation: 3.73).

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.
Not Available
Food Interactions
No interactions found.

Products

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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
VimizimSolution1 mg / mLIntravenousBiomarin International Limited2014-08-27Not applicableCanada flag
VimizimInjection, solution, concentrate5 mg/5mLIntravenousBioMarin Pharmaceutical Inc.2014-02-14Not applicableUS flag

Categories

ATC Codes
A16AB12 — Elosulfase alfa
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
ODJ69JZG85
CAS number
9025-60-9

References

General References
  1. Sanford M, Lo JH: Elosulfase alfa: first global approval. Drugs. 2014 Apr;74(6):713-8. doi: 10.1007/s40265-014-0210-z. [Article]
UniProt
P34059
KEGG Drug
D10333
PubChem Substance
347910399
RxNav
1489914
ChEMBL
CHEMBL2108676
Drugs.com
Drugs.com Drug Page
Wikipedia
Elosulfase_alfa
FDA label
Download (290 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
3CompletedTreatmentMorquio Syndrome A / MPS IVA / Mucopolysaccharidosis IV A1
3CompletedTreatmentMPS IVA1
3CompletedTreatmentMucopolysaccharidosis IVA (Morquio A Syndrome)1
2CompletedTreatmentMorquio Syndrome A / MPS IVA / Mucopolysaccharidosis IV A1
2TerminatedTreatmentMorquio Syndrome A / MPS IVA / Mucopolysaccharidosis IV A2
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
1, 2CompletedTreatmentMPS IVA1
1, 2TerminatedTreatmentMorquio Syndrome A / MPS IVA / Mucopolysaccharidosis IV A1
Not AvailableActive Not RecruitingNot AvailableMorquio Syndrome A / MPS IVA / Mucopolysaccharidosis IV Type A1
Not AvailableActive Not RecruitingNot AvailableMucopolysaccharidosis IV A1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
Injection, solution, concentrateIntravenous5 mg/5mL
Injection, solution, concentrateIntravenous; Parenteral1 MG/ML
SolutionIntravenous1 mg / mL
Injection, solutionIntravenous
SolutionIntravenous5 mg
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available

Drug created at May 06, 2015 19:53 / Updated at July 02, 2022 14:09