Alexion gets EU nod for two rare genetic disease drugs
This article was originally published in Scrip
Executive Summary
The EU's CHMP has given Alexion the marketing go-ahead for two new enzyme replacement therapies for rare genetic diseases: Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL-d); and Strensiq (asfotase alfa), the first therapy for the bone disease hypophosphatasia (HPP) that started in childhood.