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Identification
NameElosulfase alfa
Accession NumberDB09051
TypeBiotech
GroupsApproved
DescriptionElosulfase 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.
Protein structureDb09051
Related Articles
Protein chemical formulaC5020H7588N1364O1418S34
Protein average weight110800.0 Da
Sequences
>Elosulfase-alfa
APQPPNILLLLMDDMGWGDLGVYGEPSRETPLCSPSRAALLTGRLPIRNGFYTTNAHARN
LLKKAGYVSKIVGKWHLGHRPQFHPLKHGFNIPVYRDWEMVGRYYEEFPINLKTGEANLT
FLYWAVDATHAPVYASKPFLGTSQRGRYGDVADNTFVFFTSDNGAALISAPEQGGSNGPF
PGHVTAGQVSHQLGSIMDLFTTSLALAGLTLMDRPIFYYRGDTLMAATLGQHKAHFWTWT
VTTHNLEDHTKLPLIFHLGRDPGERFPLSFEALVPAQPQLNVCNWAVMNWAPPGCEKLGK
PNLDRMAAEGLLFPNFYSANAYTPQEIVGGIPDSEQLLPEDEWFGSPNCHFGPYDNKARP
QIYLQEALDFIKRQARHHPFAVREIDDSIGKILELLQDLHLCGKQTTFEGGMREPALAWW
PPSDRAIDGLNLLPTLLQGRNSWENFRQGIDFCPGQNVSGASAEYQEALSRITSVVQQHQ
CLTPPESIPKKCLWSH
Download FASTA Format
Synonyms
N-acetylgalactosamine-6-sulfatase
External Identifiers
  • BMN 110
  • J1322
  • rhGALNS
Approved Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
VimizimSolution1 mgIntravenousBiomarin International Limited2014-08-27Not applicableCanada
VimizimInjection, solution, concentrate5 mg/5mLIntravenousBio Marin Pharmaceutical Inc.2014-02-14Not applicableUs
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International BrandsNot Available
Brand mixturesNot Available
SaltsNot Available
Categories
UNIIODJ69JZG85
CAS number9025-60-9
Pharmacology
IndicationVimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Structured Indications
PharmacodynamicsAUC: 238 min x μg/mL, standard deviation 100.
Mechanism of actionMucopolysaccharidoses 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.
Related Articles
AbsorptionCmax: 1.49 μg/mL, standard deviation 0.534.
Volume of distribution

396 mL/kg, standard deviation 316.

Protein bindingNot Available
MetabolismNot Available
Route of eliminationNot Available
Half lifeweek 0: 7.52 min week 22: 35.9 min
Clearance

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

ToxicityNot Available
Affected organisms
  • Humans and other mammals
PathwaysNot Available
SNP Mediated EffectsNot Available
SNP Mediated Adverse Drug ReactionsNot Available
Interactions
Drug InteractionsNot Available
Food InteractionsNot Available
References
Synthesis ReferenceNot Available
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 ]
External Links
ATC CodesA16AB12
AHFS CodesNot Available
PDB EntriesNot Available
FDA labelDownload (290 KB)
MSDSNot Available
Pharmacoeconomics
ManufacturersNot Available
PackagersNot Available
Dosage forms
FormRouteStrength
Injection, solution, concentrateIntravenous5 mg/5mL
SolutionIntravenous1 mg
PricesNot Available
PatentsNot Available
Properties
StateSolid
Experimental PropertiesNot Available
Taxonomy
DescriptionNot Available
KingdomOrganic Compounds
Super ClassOrganic Acids
ClassCarboxylic Acids and Derivatives
Sub ClassAmino Acids, Peptides, and Analogues
Direct ParentPeptides
Alternative ParentsNot Available
SubstituentsNot Available
Molecular FrameworkNot Available
External DescriptorsNot Available

Enzymes

Kind
Protein
Organism
Human
Pharmacological action
yes
Actions
Other
General Function:
Sulfuric ester hydrolase activity
Specific Function:
Not Available
Gene Name:
GALNS
Uniprot ID:
P34059
Molecular Weight:
58025.575 Da
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 ]
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Drug created on May 06, 2015 13:53 / Updated on August 17, 2016 12:24