Alexion's hypophosphatasia drug asfotase alfa under FDA review
This article was originally published in Scrip
Executive Summary
Alexion Pharmaceuticals said it has completed its rolling biologics license application submission to the FDA for asfotase alfa as a treatment for hypophosphatasia (HPP), a chronic and progressive ultra-rare metabolic disease, which can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.