Sebelipase Alfa
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.
Etki Mekanizması
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)
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.
Sıkça Sorulan Sorular
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
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3039537. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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