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Cipaglucosidase Alfa

CHEMBL4594329 Phase 4 Onaylandı Enzyme
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A next-generation recombinant acid alpha-glucosidase enzyme replacement therapy designed with improved targeting to muscle lysosomes via the mannose-6-phosphate receptor for the treatment of Pompe disease, a rare lysosomal storage disorder. It is used with miglustat as a stabilizer to improve cellular uptake and efficacy compared to earlier enzyme replacement options. This combination provides improved clinical outcomes including better muscle function and respiratory status.

Etki Mekanizması

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mekanizma

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

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.

Sıkça Sorulan Sorular

A next-generation recombinant acid alpha-glucosidase enzyme replacement therapy designed with improved targeting to muscle lysosomes via the mannose-6-phosphate receptor for the treatment of Pompe disease, a rare lysosomal storage disorder. It is used with miglustat as a stabilizer to improve cellular uptake and efficacy compared to earlier enzyme replacement options. This combination provides improved clinical outcomes including better muscle function and respiratory status.

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

Yes, Cipaglucosidase 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). CHEMBL4594329. Open-access bioactivity database.

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

Tıbbi Sorumluluk Reddi

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.