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Alexion Snags 2nd FDA Voucher On Kanuma Win

This article was originally published in Scrip

Executive Summary

The FDA's approval on Dec. 8 of Alexion Pharmaceuticals Inc.'s enzyme replacement therapy (ERT) Kanuma (sebelipase alfa) was significant for several reasons – mostly, because it provides patients with the first treatment in the US for a deadly, progressive ultra-rare genetic disease, known as lysosomal acid lipase deficiency (LAL-D), which causes multi-organ damage, resulting in premature death.