A Synthetic Review of Eliglustat, a Drug for Gaucher’s Disease

Gaucher's disease (GD) is a rare genetic disorder that falls under the category of lysosomal storage diseases (LSDs) and is caused by the defective production of the lysosomal enzyme glucocerebrosidase. As a result, glucosylceramide (GlcCer) is not hydrolyzed into glucose and ceramide, which causes GlcCer to build up in the macrophages. Births with GD are recorded at a rate of 1/40,000 to 1/60,000 in the general population, but in the case of the Ashkenazi Jewish community, this rate goes up to 1/800 births. The first oral medication for the treatment of GD was miglustate. Then a new oral medication, eliglustat (1), was introduced to treat GD type 1. In this review, we explore various synthetic approaches to synthesizing 1, which will assist in designing newer approaches.