name: | SebelipaseAlfa |
ATC code: | A16AB14 | route: | intravenous |
n-compartments | 2 |
Sebelipase alfa is a recombinant human lysosomal acid lipase enzyme replacement therapy indicated for the treatment of lysosomal acid lipase deficiency (LAL-D), a rare genetic disorder causing the accumulation of cholesteryl esters and triglycerides. It is approved for use in both pediatric and adult patients with LAL-D.
Pharmacokinetic parameters reported for adult and pediatric (≥8 years) patients with LAL-D receiving intravenous sebelipase alfa at a dose of 1 mg/kg; values are geometric mean population PK model parameters from the published study.