Alexion Pharma Reports Data From Phase 2 Study Of Asfotase Alfa - Quick Facts

(RTTNews) - Alexion Pharmaceuticals Inc. (ALXN) reported data from an ongoing multinational Phase 2 study of asfotase alfa in infants and young children with hypophosphatasia, or HPP, an inherited, ultra-rare metabolic disorder. The study met its primary endpoint: infants and young children with HPP treated with asfotase alfa had significant improvement in skeletal mineralization from baseline as assessed radiographically after 24 weeks of treatment. This response was observed as early as 12 weeks and improvement continued at 48 weeks, the company said.

Ninety three percent of the patients survived the first 48 weeks of treatment with 80% of patients having improved respiratory status or requiring no respiratory support at the final analysis.