Eliglustat is intended to be used for the treatment of type 1 Gaucher disease in treatment naïve and treatment experienced patients. If licensed, it has the potential to improve the quality of life of patients through the improved treatment convenience of oral administration. Eliglustat is an oral glucosylceramide analogue that inhibits glucosylceramide synthase, resulting in reduced production of glucosylceramide ̶ the substance which accumulates in the tissues and cells of Gaucher disease patients. It is not licensed for any other indication.
Gaucher disease is a rare condition and the most prevalent of the lysosomal storage disorders. Over 90% of affected individuals have type 1 Gaucher disease with high frequencies particularly seen in people of Eastern European (Ashkenazi) Jewish decent. Prevalence of type 1 Gaucher disease has been estimated to be 1 in 200,000 (non-Ashkenazi…
