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Cerliponase Alfa

CHEMBL3544921 Phase 4 承認済み Enzyme
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A recombinant human tripeptidyl peptidase 1 enzyme administered directly into the brain via intracerebroventricular infusion to treat neuronal ceroid lipofuscinosis type 2 (CLN2 disease), a rare and fatal inherited neurological disorder in children. It replaces the enzyme that is deficient in affected patients, slowing the neurological deterioration characteristic of the disease. It is the first enzyme replacement therapy approved for direct central nervous system administration.

作用機序

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

Pharmacokinetics (PK)

Pharmacodynamics (PD)

機序

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.

よくある質問

A recombinant human tripeptidyl peptidase 1 enzyme administered directly into the brain via intracerebroventricular infusion to treat neuronal ceroid lipofuscinosis type 2 (CLN2 disease), a rare and fatal inherited neurological disorder in children. It replaces the enzyme that is deficient in affected patients, slowing the neurological deterioration characteristic of the disease. It is the first enzyme replacement therapy approved for direct central nervous system administration.

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

Yes, Cerliponase 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). CHEMBL3544921. Open-access bioactivity database.

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

医学的免責事項

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.