FDA grants priority review to Alexion's ERT asfotase alfa
This article was originally published in Scrip
Executive Summary
The FDA has granted a priority review to Alexion's enzyme replacement therapy asfotase alfa as a treatment for patients with infantile- and juvenile-onset hypophosphatasia (HPP), a chronic and progressive ultra-rare genetic metabolic disease characterized by defective bone mineralization, which can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.