Sebelipase alfa (Kanuma©) helps maintain initial improvements in liver and cholesterol parameters over the long term for patients with lysosomal acid lipase deficiency (LAL-D). With no worsening liver disease, this treatment helps patients aged 4 years and older for up to 5 years.

This is according to the final results of the phase 3 clinical trial called ARISE, which evaluated the safety and efficacy of 1 mg/kg of intravenous sebelipase alfa infusions administered every other week in patients with late-onset LAL-D.

The trial had a 20-week, double-blind, placebo-controlled phase, a 130-week, open-label extension phase, and a 104-week, open-label expanded treatment phase. All participants received the treatment in the open-label phases.

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The primary outcome measure was the percentage of participants achieving alanine aminotransferase (ALT) normalization.

Secondary outcome measures included percent change in low, nonhigh, and high-density lipoprotein cholesterol; triglycerides, liver fat content, and liver volume from baseline; the percentage of participants achieving aspartate aminotransferase (AST) normalization; and the number of participants with improvement in liver histopathology.

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At the last open-label visit, ALT levels had normalized in 47% and AST levels had normalized in 66% of patients. Patients who switched from placebo to sebelipase alfa had sustained improvements in ALT and AST levels during the open-label phases of the study. There was a 25% reduction in low-density lipoprotein cholesterol and a 27% increase in high-density lipoprotein cholesterol.

During the open-label phases, most adverse events were mild to moderate in severity. Of 66 patients who were enrolled in the study, 13 had infusion-associated reactions, 1 of which was serious, and 6 patients developed antibodies against the treatment.

Sebelipase alfa is an enzyme replacement therapy for LAL-D. It is a recombinant form of the lysosomal acid lipase enzyme that is defective in LAL-D. The US Food and Drug Administration (FDA) has approved it for patients of all ages.


Burton BK, Feillet F, Furuya KN, Marulkar S, Balwani M. Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: final results of the ARISE study. J Hepatol. Published online November 10, 2021. doi:10.1016/j.jhep.2021.10.026

Acid lipase replacement investigating safety and efficacy (ARISE) in participants with lysosomal acid lipase deficiency (ARISE). US National Library of Medicine. Last updated: December 29, 2020. Accessed November 12, 2021.