Alexion’s late stage orphan drug granted “breakthrough drug” designation

Alexion Pharmaceuticals, manufacturers of blockbuster orphan drug Soliris, has announced that the US Food and Drug Administration (FDA) has granted a Breakthrough Therapy designation to a separate late-stage orphan drug in Alexion's pipeline (press release). The drug, called asfotase alfa, is a first-in-class targeted enzyme replacement therapy for the treatment of patients with hypophosphatasia (HPP). HPP is an ultra-rare metabolic …