- The Japanese Ministry of Health, Labour and Welfare approves Genzyme's (SNY +0.4%) Cerdelga (eliglustat) for the oral treatment of patients with Gaucher disease type 1.
- Gaucher disease affects fewer than 10,000 people worldwide. It is characterized by an insufficient amount of an enzyme, glucocerebrosidase, that breaks down a certain type of fat molecule. As a results, lipid engorged cells (Gaucher cells) build up in different parts of the body, principally the spleen, liver and bone marrow. The accumulation causes spleen and liver enlargement, anemia, excessive bleeding and bruising and bone disease. Cerdelga inhibits the enzyme glucosylceramide synthase, which reduces the production of the offending fat molecule glucosylceramide.
- Cerdelga was approved by the FDA in August 2014, the European Commission in January 2015 and the Australian Therapeutic Goods Administration in February 2015.