💊 Médicaments A-Z 🧬 Cibles 🛠 Outils 📚 Guides
Tous les médicaments Classes de médicaments Aires thérapeutiques Effets Indésirables
Guides Glossaire Chronologie
Comparaisons Parcours des médicaments Familles de médicaments
🇺🇸 English 🇰🇷 한국어 🇯🇵 日本語 🇨🇳 中文简体 🇪🇸 Español 🇧🇷 Português 🇮🇳 हिन्दी 🇸🇦 العربية 🇫🇷 Français 🇷🇺 Русский 🇩🇪 Deutsch 🇹🇷 Türkçe 🇻🇳 Tiếng Việt 🇮🇩 Bahasa Indonesia 🇹🇭 ไทย

Sebelipase Alfa

CHEMBL3039537 Phase 4 Approuvé Enzyme
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Sebelipase alfa is a recombinant lysosomal acid lipase (LAL) enzyme replacement therapy used for the treatment of lysosomal acid lipase deficiency (LAL-D), including the rapidly fatal Wolman disease in infants and the more slowly progressive cholesteryl ester storage disease (CESD). The enzyme hydrolyzes cholesteryl esters and triglycerides within lysosomes, preventing lipid accumulation in hepatocytes and other cells. It has demonstrated dramatic improvement in survival in infantile-onset LAL-D and reversal of liver pathology in older patients.

Mécanisme d'action

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)

Mécanisme

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.

Foire aux questions

Sebelipase alfa is a recombinant lysosomal acid lipase (LAL) enzyme replacement therapy used for the treatment of lysosomal acid lipase deficiency (LAL-D), including the rapidly fatal Wolman disease in infants and the more slowly progressive cholesteryl ester storage disease (CESD). The enzyme hydrolyzes cholesteryl esters and triglycerides within lysosomes, preventing lipid accumulation in hepatocytes and other cells. It has demonstrated dramatic improvement in survival in infantile-onset LAL-D and reversal of liver pathology in older patients.

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, Sebelipase 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). CHEMBL3039537. Open-access bioactivity database.

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

Avertissement médical

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.