Alexion Pharmaceuticals reports that Japan approved a New Drug Application for the use of Strensiq® (asfotase alfa) as a treatment for patients in Japan with hypophosphatasia (HPP), a life-threatening, ultra-rare metabolic disorder. Strensiq, a bone-targeted enzyme replacement therapy, is the first therapy approved in Japan for the treatment of patients with HPP … more

Earlier Alexion reported that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted positive opinions recommending marketing authorization of Strensiq™ and Kanuma™ (sebelipase alfa). The proposed indication for Kanuma is for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency … more