FDA approves Alexion's Kanuma for LAL-D

FDA approved Kanuma sebelipase alfa from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) to treat lysosomal acid lipase deficiency and granted the company a Priority Review voucher under the agency's rare pediatric disease Priority Review voucher program. The agency approved Kanuma to treat both Wolman disease, the form of LAL-D that presents in infants, and cholesteryl ester storage disease (CESD), a later-onset form of LAL-D.

Alexion said it will launch Kanuma during the first week of January. On a conference call Tuesday, EVP and CFO Vikas Sinha said the drug's average annual cost will be $310,000, based on patients' average weight, age and other factors. He said he anticipates a "slow and steady" launch trajectory. ...