Alexion reports asfotase alfa Phase II data

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) said subcutaneous asfotase alfa met the primary endpoint in the Phase II portion of a Phase II/III trial to treat hypophosphatasia (HPP) in 13 patients five years of age and younger. Asfotase alfa ( ENB-0040) significantly improved skeletal mineralization as measured by Radiographic Global Impression of Change (RGI-C) scale scores at week 24 compared to baseline (p=0.001). Data were presented at the Pediatric Endocrinology meeting in Milan. Alexion plans to enroll about 60 patients in the trial, with final data expected next year. The company plans to submit a BLA to FDA and an MAA to EMA in mid-2014 for asfotase alfa to treat HPP in patients whose first signs or symptoms occurred prior to 18 years of age. ...