Wolman Disease

October 17, 2016

Alexion Pharmaceuticals released results of an ongoing, open-label extension of the pivotal phase III study for Kanuma (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D), also called Wolman disease. At 52 weeks, nearly all patients (97%) who had received Kanuma from the start of the double-blind period had a rapid and sustained reduction in alanine aminotransferase (ALT), with a mean percent reduction of 53%, and an increase from 31% (11/36) to 45% of patients (15/33) achieving ALT normalization. Similarly, after 52 weeks of Kanuma treatment, nearly all patients (97%) who had initially received placebo during the double-blind period had a reduction in ALT, with a mean percent reduction of 52%, and 48% of patients (14/29) achieving ALT normalization. Sustained improvements were also observed in both groups in markers of lipid abnormalities (including LDL cholesterol, non-HDL cholesterol, triglycerides and HDL cholesterol) through 52 weeks of Kanuma treatment. The safety profile of Kanuma during the extended open-label period was consistent with that observed in the double-blind period. Most treatment-emergent adverse events (TEAEs) were mild to moderate in severity, and no patient discontinued the open-label study because of adverse events. The most common TEAEs in the open-label period were headache (40%), nasopharyngitis (35%), and cough (28%). Twelve patients (18%) experienced an infusion-associated reaction during the open-label period; all but one reaction were mild or moderate in intensity.