Elosulfase alfa

Identification

Name
Elosulfase alfa
Accession Number
DB09051
Description

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
Db09051
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
  • N-acetylgalactosamine-6-sulfatase
External IDs
  • 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).

Associated Conditions
Contraindications & Blackbox Warnings
Learn about our commercial Contraindications & Blackbox Warnings data.
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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
Affected organisms
  • Humans and other mammals
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
Not Available

Products

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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
VimizimInjection, solution, concentrate5 mg/5mLIntravenousBioMarin Pharmaceutical Inc.2014-02-14Not applicableUS flag
VimizimSolution1 mgIntravenousBiomarin International Limited2014-08-27Not applicableCanada flag
Additional Data Available
  • Application Number
    Application Number
    Available for Purchase

    A unique ID assigned by the FDA when a product is submitted for approval by the labeller.

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  • Product Code
    Product Code
    Available for Purchase

    A governmentally-recognized ID which uniquely identifies the product within its regulatory market.

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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

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. [PubMed:24700469]
UniProt
P34059
KEGG Drug
D10333
PubChem Substance
347910399
RxNav
1489914
ChEMBL
CHEMBL2108676
Drugs.com
Drugs.com Drug Page
Wikipedia
Elosulfase_alfa
AHFS Codes
  • 44:00.00 — Enzymes
FDA label
Download (290 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
3CompletedTreatmentMorquio A Syndrome / MPS IVA / Mucopolysaccharidosis IV A1
3CompletedTreatmentMPS IV A1
3CompletedTreatmentMucopolysaccharidosis IVA (Morquio A Syndrome)1
2CompletedTreatmentMorquio A Syndrome / MPS IVA / Mucopolysaccharidosis IVA1
2TerminatedTreatmentMorquio A Syndrome / MPS IVA / Mucopolysaccharidosis IVA2
1Not Yet RecruitingTreatmentGaucher Disease, Type 2 / Gaucher Disease, Type 3 / MPS I / MPS II / MPS IVA / MPS VI / MPS VII / Pompe Disease Infantile-Onset / Wolman's Disease1
1, 2CompletedTreatmentMPS IV A1
1, 2TerminatedTreatmentMorquio A Syndrome / MPS IV A / Mucopolysaccharidosis IVA1
Not AvailableApproved for MarketingNot AvailableMorquio A Syndrome / MPS IVA / Mucopolysaccharidosis IVA1
Not AvailableRecruitingNot AvailableMorquio A Syndrome / MPS IVA / Mucopolysaccharidosis IV Type A1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
Injection, solution, concentrateIntravenous5 mg/5mL
Injection, solution, concentrateIntravenous; Parenteral1 MG/ML
SolutionIntravenous1 mg
Injection, solutionIntravenous1 mg/ml
SolutionIntravenous5 mg
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available

Drug created on May 06, 2015 13:53 / Updated on November 29, 2020 03:54

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