CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) to asfotase alfa for the treatment of patients with hypophosphatasia (HPP), a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization. Patients with HPP can experience a range of devastating consequences, including the destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.1-5 Asfotase alfa is an investigational, highly innovative, first-in-class targeted enzyme replacement therapy designed to address the underlying cause of HPP.
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