vestronidase alfa 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
enzymes	5264	1638194-78-1

Description:

Molecule	Description
Molfile Synonyms: vestronidase alfa vestronidase alfa-vjbk mepsevii UX003	Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues. Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Molfile

Synonyms:

vestronidase alfa
vestronidase alfa-vjbk
mepsevii
UX003

Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.

Molecular weight:
Formula:
CLOGP:
LIPINSKI: None
HAC: None
HDO: None
TPSA:
ALOGS:
ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

Date	Agency	Company
Aug. 23, 2018	EMA	ULTRAGENYX PHARM INC
Nov. 15, 2017	FDA	ULTRAGENYX PHARM INC
Jan. 20, 2022	PMDA	AMICUS THERAPEUTICS, INC.

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	A16AB18	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Enzymes
FDA EPC	N0000193804	Lysosomal beta Glucuronidase

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Mucopolysaccharidosis, MPS-VII	indication	43916004	DOID:12803

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

None

External reference:

ID	Source
7XZ4062R17	UNII
4037140	VANDF
4037141	VANDF
C4519728	UMLSCUI
CHEMBL3707382	ChEMBL_ID
DB12366	DRUGBANK_ID
D11004	KEGG_DRUG
10305	INN_ID
266336	MMSL
33275	MMSL
33399	MMSL
d08681	MMSL
017418	NDDF
763609003	SNOMEDCT_US
763610008	SNOMEDCT_US
1989818	RXNORM
C000654126	MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
MEPSEVII	HUMAN PRESCRIPTION DRUG LABEL	1	69794-001	INJECTION	2 mg	INTRAVENOUS	BLA	25 sections
MEPSEVII	HUMAN PRESCRIPTION DRUG LABEL	1	69794-001	INJECTION	2 mg	INTRAVENOUS	BLA	25 sections