Asfotase Alfa Significantly Decreased TNSALP Substrates and Improved 6-Minute Walk Test Results in Adolescents and Adults with Hypophosphatasia

Alexion Pharmaceuticals, Inc. ALXN today announced data from a Phase 2 study of asfotase alfa in adolescents and adults with hypophosphatasia (HPP), a severe and ultra-rare metabolic disorder. In the study, all patients who were treated with asfotase alfa had an objective response to therapy as indicated by a reduction in tissue non-specific alkaline phosphatase (TNSALP) substrates. In addition, treated patients demonstrated improvement in the six-minute walk test (6MWT). Asfotase alfa is an investigational therapy being developed by Alexion as a potential treatment for patients with HPP. Data were presented today at the American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting, being held March 27-31 in Charlotte, NC.