Lysosomal acid lipase (LAL) deficiency

Active Ingredient: Sebelipase alfa

Indication for Sebelipase alfa

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.

For this indication, competent medicine agencies globally authorize below treatments:

1 mg/kg once every other week

Route of admnistration


Defined daily dose

1 - 1 mg per kg of body weight

Dosage regimen

From 1 To 1 mg per kg of body weight once every 14 day(s)

Detailed description

The recommended dose in children and adults who do not present with rapidly progressive LAL deficiency prior to 6 months of age is 1 mg/kg administered as an intravenous infusion once every other week.

Dosage considerations

The total volume of the infusion should be administered over approximately 2 hours. A 1-hour infusion may be considered after patient tolerability is established. The infusion period may be extended in the event of dose escalation.

Active ingredient

Sebelipase alfa

Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Replacement of LAL enzyme activity leads to reductions in liver fat content and transaminases, and enables metabolism of cholesteryl esters and triglycerides in the lysosome, leading to reductions in lowdensity lipoprotein (LDL) cholesterol and non-high-density lipoprotein (HDL) cholesterol, triglycerides, and increases in HDL cholesterol. Improvement in growth occurs as a result of substrate reduction in the intestine.

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