1 September 2015 - Alexion Pharmaceuticals, Inc. announced today that the European Commission (EC) has approved Kanuma (sebelipase alfa) for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase deficiency (LAL-D). Kanuma, an innovative ERT, is the first approved treatment in the European Union for patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death. Alexion expects to begin serving patients in Germany in October and is now commencing reimbursement processes with healthcare authorities in each of the major European countries.

“Today’s approval is a crucial milestone for patients with LAL-D, a grave condition that can have devastating consequences for patients of all ages,” said Vassili Valayannopoulos, M.D., Ph.D., investigator in the Kanuma pivotal studies, Hôpital Necker-Enfants Malades and IMAGINE Institute, Paris. “In clinical studies, 67% of infants treated with Kanuma survived beyond 12 months of age, whereas without treatment, these patients would have faced a near-certain fatal outcome. In pediatric and adult patients, Kanuma was also shown to reduce the markers of liver injury and lipid accumulation, which can lead to serious and life-threatening complications.”

For more details, go to: http://news.alexionpharma.com/press-release/product-news/european-commission-grants-marketing-authorization-kanuma-sebelipase-alfa