Alexion reports long-term data from extension of Phase 3 Kanuma study
Alexion Pharma announced that researchers presented new long-term data from an ongoing, open-label extension of the pivotal Phase 3 ARISE trial of Kanuma in children and adults with lysosomal acid lipase deficiency. At 52 weeks of Kanuma treatment, nearly all patients -- 97 percent -- who had received Kanuma from the start of the double-blind period had a rapid and sustained reduction in alanine aminotransferase, with a mean percent reduction of 53 percent, and an increase from 31 percent to 45 percent of patients achieving ALT normalization. Similarly, after 52 weeks of Kanuma treatment, nearly all patients -- 97 percent -- who had initially received placebo during the double-blind period had a reduction in ALT, with a mean percent reduction of 52 percent, and 48 percent of patients 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. No patient discontinued the open-label study because of adverse events. The data were reported at the World Congress of Pediatric Gastroenterology, Hepatology and Nutrition.