Alexion Pharmaceuticals Inc Announces Data From Phase 2 Study Of Asfotase Alfa In Hypophosphatasia

Alexion Pharmaceuticals Inc announced that asfotase alfa (formerly known as ENB-0040), a investigational targeted enzyme replacement therapy, was shown to improve skeletal abnormalities, pulmonary and physical function, and cognitive development in a Phase 2 study of infants and young children with life-threatening hypophosphatasia (HPP). Alexion is developing asfotase alfa as a potential treatment for patients with HPP, an ultra-rare, genetic, life-threatening metabolic disease for which there are currently no approved or effective treatment options. Due to a genetic enzyme deficiency, symptomatic patients with HPP face progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure.1,2,3,4 About half of newborns with HPP do not survive past one year of age.