Data Presented at WORLDSymposium™ 2018 Show Survival to 3 Years of Age and Improvements in Liver Function in Infants with Lysosomal Acid Lipase Deficiency Treated with Kanuma® (sebelipase ...
Alexion Pharmaceuticals, Inc (ALXN)
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Source: Business Wire
NEW HAVEN, Conn.--(BUSINESS WIRE)-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the combined interim data from two ongoing open-label studies, VITAL and CL-08, show a 3-year survival estimate of 68% in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D) treated with Kanuma® (sebelipase alfa).1 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.1 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.2,3 “Rapidly progressive LAL-D previously meant a death sentence for
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