Three year survival data from trial of Kanuma in infants with lysosomal acid lipase deficiency.- Alexion.

Alexion Pharmaceuticals, Inc. announced that the combined interim data from two ongoing open-label studies VITAL study and CL-08 study show a 3-year survival estimate of 68% in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D) treated with Kanuma (sebelipase alfa). Of the 19 infants who were enrolled in the studies, 7 infants are surviving and have reached 3 years of age while an additional 6 infants have not yet reached 3 years of age.

These infants also benefited from improvements in a number of key parameters including weight gain and markers of liver disease and function. There were no discontinuations due to adverse events. These data were presented at the WORLDSymposium in San Diego and confirm and extend previously published survival data from these studies.