Vestronidase alfa is a recombinant beta-glucuronidase used to treat mucopolysaccharidosis VII. Vestronidase alfa, or vestronidase alfa-vjbk, is a recombinant human lysosomal beta glucuronidase that is a purified enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line.
- Mechanism
- Curator reviewed · 1 reference
Beta-glucuronidase (GUS) is a lysosomal enzyme responsible for degradation of glucuronate-containing glycosaminoglycan (GAG). Resulting lysosomal storage and GAG accumulation in cells from incomplete metabolic degradation of macromolecules leads to damage to multiple tissues and organs. Vestronidase alfa serves as an exogenous source of GUS enzyme for uptake into cellular lysosomes, which is facilitated by the presence of mannose-6-phosphate (M6P) residues on the oligosaccharide chains of the recombinant enzyme. The chains allow binding of the enzyme to cell surface receptors to promote cellular uptake, and targets the lysosomes to achieve catabolism of accumulated GAGs in affected tissues.
- Primary indication
- Indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).Curator reviewed · 1 structured indication
- Formula / weight
- 72562.0 Da (Non-glycosylated)
- First approval
- United States, 2017 · European Union, 2020
- Also known as
- Recombinant human beta-glucuronidase · Vestronidase alfa-vjbk
- Code names
- UX-003
- Brand names
Resolves to
What you can answer from here — as of February 04, 2025
Which drugs share a target with Vestronidase alfa, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account