Cerdelga update
Following the Food and Drug Administration (FDA) approval of Cerdelga as an oral treatment for certain adult Gaucher disease type 1 patients in August, press reports suggest that Genzyme has set a price for the US patients at $310,250 a year, see press release: http://www.bostonglobe.com/business/2014/09/02/new-genzyme-pill-treat-rare-gaucher-disease-will-cost-patients-year/5thkIb587nKi7zRAb9GgxM/story.html
In Europe, marketing applications for Cerdelga are under review by the European Medicines Agency (EMA) and other regulatory authority, a decision is expected at the end of 2014.