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Kanuma reduces liver fat in lysosomal acid lipase deficiency

In a new study, researchers found that therapy with Kanuma, also known as sebelipase alfa, reduced hepatic and lipid abnormalities among adults and children with lysosomal acid lipase deficiency, according to published findings.

“This study is important because it demonstrates that sebelipase alfa can reduce fat accumulation in the liver in patients with [lysosomal acid lipase deficiency] with subsequent improvement or normalization of alanine aminotransferase,” Barbara K. Burton, MD, professor in pediatrics-genetics, birth defects and metabolism, Northwestern University Feinberg School of Medicine, told Healio.com/Hepatology. “This should improve the outcome of this disorder which, untreated, leads to cirrhosis and liver transplantation.” 

Barbara K. Burton

Burton and colleagues randomly assigned 66 patients with lysosomal acid lipase deficiency (LALD) to an intravenous infusion of 1 mg per kg of body weight dosage of Kanuma (sebelipase alfa, Alexion; n = 36), or placebo (n = 30) every other week for 20 weeks. After the double-blind treatment period, patients entered an open-label extension period where they all received sebelipase alfa.

Overall, 65 patients completed the double-blind portion of the trial and continued with open-label treatment. At 20 weeks, the ALT level was normal in 31% of patients who received sebelipase alfa (n = 11) and 7% of patients in the placebo group (n = 30; P = .03).

Compared to baseline, patients in the sebelipase alfa group had a mean change in ALT of – 58 U per liter compared with patients who received placebo and had a mean change of – 7 U per liter (P < .001).

The researchers observed improvements in lipid levels and reduction in hepatic fat content (P < .001 for all comparisons) except for triglycerides (P = .04), according to the research.

The number of patients who experienced adverse events was similar for both groups. A majority of adverse events were mild and considered unrelated to treatment. Three serious adverse events were reported during the double-blind period, of which two were in the sebelipase alfa group. One of the events in this group was considered related to the study drug, according to the research. No serious adverse events occurred during the open-label period that were related to the study drug.

“With the availability of sebelipase alfa, LALD is now a treatable cause of cirrhosis,” Burton said. “It also improves the secondary dyslipidemia associated with the disorder, reducing the risk of cardiovascular disease.” – by Melinda Stevens  

Disclosures: Burton reports receiving consulting fees from BioMarin Pharmaceutical, Shire, REGENXBIO and Hyperion Therapeutics; lecture fees from Genzyme and Shire; and grant support from Genzyme, BioMarin Pharmaceutical, Shire, Synageva BioPharma, Ultragenyx Pharmaceutical and Cytonet. Please see the study for a full list of all other authors’ relevant financial disclosures.