Unsurprising FDA 'breakthrough' designation for Alexion's hypophosphatasia drug
This article was originally published in Scrip
Executive Summary
Wall Street was not surprised on 28 May by the US FDA granting Alexion Pharmaceuticals' investigational hypophosphatasia (HPP) drug asfotase alfa (ENB-0040) a breakthrough therapy designation, given the high unmet medical need for the potentially life-threatening, ultra-rare metabolic genetic disease.