The Food and Drug Administration (FDA) has approved Kanuma (sebelipase alfa) for the treatment of patients with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease. Kanuma is manufactured by Alexion Pharmaceuticals.

This is the first treatment approved in the U.S. for this condition. It is an enzyme replacement therapy that reduces substrate accumulation in the lysosomes of cells throughout the body. Sebelipase alfa binds to cell surface receptors via glycans expressed in the protein and is subsequently internalized into lysosomes. It catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol and free fatty acids.

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The approval of Kanuma was based on data from two studies and a supporting open–label extension study that included infant, pediatric, and adult patients with LAL-D. Results indicated significant benefit in terms of survival (67%) in patients with infant form of LAL-D beyond 12 months vs. 0% survival among patients that were untreated. Treatment with Kanuma also led to larger reductions from baseline in ALT values and liver fat content vs. the placebo. 

Kanuma will be available as 20mg/10mL strength preservative-free solution in single-use vials. It is expected to launch during the first week of January 2016.

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