Medical Uses
Kanuma (sebelipase alfa), from Alexion Pharmaceuticals (AstraZeneca Rare Disease), is indicated for the treatment of patients with Lysosomal Acid Lipase Deficiency (LAL-D), including infantile-onset (Wolman disease) and later-onset forms.
LAL-D is caused by a deficiency of the lysosomal acid lipase enzyme, leading to abnormal buildup of fats (cholesteryl esters and triglycerides) in cells of the liver, spleen, and other organs. Sebelipase alfa is a recombinant form of this enzyme, replacing the missing/deficient enzyme to help break down the accumulated lipids.
