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| Stem definition | Drug id | CAS RN |
|---|---|---|
| enzymes | 5264 | 1638194-78-1 |
| Molecule | Description |
|---|---|
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Synonyms:
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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.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 23, 2018 | EMA | ULTRAGENYX PHARM INC | |
| Nov. 15, 2017 | FDA | ULTRAGENYX PHARM INC | |
| Jan. 20, 2022 | PMDA | AMICUS THERAPEUTICS, INC. |
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| 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 |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Mucopolysaccharidosis, MPS-VII | indication | 43916004 | DOID:12803 |
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| ID | Source |
|---|---|
| 7XZ4062R17 | UNII |
| 4037140 | VANDF |
| 4037141 | VANDF |
| C4519728 | UMLSCUI |
| CHEBI:755838 | CHEBI |
| CHEMBL3707382 | ChEMBL_ID |
| DB12366 | DRUGBANK_ID |
| D11004 | KEGG_DRUG |
| 10305 | INN_ID |
| C000654126 | MESH_SUPPLEMENTAL_RECORD_UI |
| 763609003 | SNOMEDCT_US |
| 763610008 | SNOMEDCT_US |
| 266336 | MMSL |
| 33275 | MMSL |
| 33399 | MMSL |
| d08681 | MMSL |
| 017418 | NDDF |
| 1989818 | RXNORM |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| MEPSEVII | HUMAN PRESCRIPTION DRUG LABEL | 1 | 69794-001 | INJECTION | 2 mg | INTRAVENOUS | BLA | 25 sections |