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Vestronidase Alfa

CHEMBL3707382 Phase 4 Zugelassen Enzyme
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Vestronidase alfa is a recombinant human lysosomal enzyme (beta-glucuronidase, GUS) used for enzyme replacement therapy in patients with mucopolysaccharidosis type VII (MPS VII, Sly syndrome), a rare lysosomal storage disease caused by GUS deficiency leading to glycosaminoglycan accumulation. Administered intravenously, it is taken up by cells via mannose-6-phosphate receptors and reduces the lysosomal storage of heparan sulfate, dermatan sulfate, and chondroitin sulfate. It is the only approved treatment for this ultra-rare lysosomal storage disorder.

Wirkmechanismus

As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mechanismus

As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Häufig gestellte Fragen

Vestronidase alfa is a recombinant human lysosomal enzyme (beta-glucuronidase, GUS) used for enzyme replacement therapy in patients with mucopolysaccharidosis type VII (MPS VII, Sly syndrome), a rare lysosomal storage disease caused by GUS deficiency leading to glycosaminoglycan accumulation. Administered intravenously, it is taken up by cells via mannose-6-phosphate receptors and reduces the lysosomal storage of heparan sulfate, dermatan sulfate, and chondroitin sulfate. It is the only approved treatment for this ultra-rare lysosomal storage disorder.

As a recombinant protein, it replicates the function of the naturally occurring human protein, supplementing or replacing deficient endogenous production to restore normal physiological activity.

Yes, Vestronidase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3707382. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

Medizinischer Haftungsausschluss

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.