The FDA has accepted for priority review Alexion’s application for asfotase alfa, an investigational, first-in-class enzyme replacement therapy for treatment of patients with infantile- and juvenile-onset hypophosphatasia.

“If approved, asfotase alfa will be the first treatment for patients with HPP, a devastating disease that can result in impaired respiratory function, severe disability and premature death for some patients,” said Leonard Bell, M.D., Chairman and Chief Executive Officer of Alexion … more

New data from a retrospective, multinational natural history study of children with hypophosphatasia were presented in a late-breaking oral session at an Endocrine Society meeting in San Diego … more