Elosulfase alfa
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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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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Management of Mucopolysaccharidosis type iva •••••••••••• - 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
- 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 Vimizim Solution 1 mg / mL Intravenous Biomarin International Limited 2014-08-27 Not applicable Canada Vimizim Injection, solution, concentrate 5 mg/5mL Intravenous BioMarin Pharmaceutical Inc. 2014-02-14 Not applicable US
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
- Sanford M, Lo JH: Elosulfase alfa: first global approval. Drugs. 2014 Apr;74(6):713-8. doi: 10.1007/s40265-014-0210-z. [Article]
- External Links
- UniProt
- P34059
- KEGG Drug
- D10333
- PubChem Substance
- 347910399
- 1489914
- ChEMBL
- CHEMBL2108676
- Drugs.com
- Drugs.com Drug Page
- Wikipedia
- Elosulfase_alfa
- FDA label
- Download (290 KB)
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 3 Completed Treatment Mucopolysaccharidosis Type IVA 3 2 Completed Treatment Mucopolysaccharidosis Type IVA 1 2 Terminated Treatment Mucopolysaccharidosis Type IVA 2 1 Recruiting Treatment Gaucher Disease, Type II / Gaucher Disease, Type III / Infantile-onset Pompe Disease / MPS VI / Mucopolysaccharidosis Type II (MPS II) / Mucopolysaccharidosis Type IVA / Mucopolysaccharidosis Type VII / Myocardial Perfusion Imaging / Wolman's Disease 1 1, 2 Completed Treatment Mucopolysaccharidosis Type IVA 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Injection, solution, concentrate Intravenous 5 mg/5mL Injection, solution, concentrate Intravenous; Parenteral 1 MG/ML Solution Intravenous 1 mg / mL Injection, solution Intravenous Solution Intravenous 5 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