- Cerdelga (eliglustat), which is intended for long-term use in patients with type 1 Gaucher disease, has been approved by the European Commission, after being approved in the U.S. last August.
- Cerdelga can be used in patients with Gaucher disease who are CYP2D6 poor metabolizers, intermediate metabolizers, or extensive metabolizers.
- Cerdelga is a orally available glucosylceramide analog, which partially inhibits the enzyme glucosylceramide, with the end result being that glucosylceramide does not build up in the tissues and muscles of patients who normally face this problem.
With the introduction of Cerdelga, Genzyme (an arm of Sanofi) is continuing its commitment to rare diseases, including Gaucher, a rare genetic disease.
In 1985, Genzyme introduced Ceredase, which is still prescribed by doctors today. Gaucher disease affects a small part of the population (20,000 as of the mid-1990's). However, it can be treated with enzyme replacement—a process that is constantly being improved based on Genzyme's efforts in this therapeutic area.