Sanofi S.A. is a French multinational pharmaceutical company headquartered in Paris, with a significant investment in rare disease therapies and lysosomal storage disorders. Sanofi's interest in the ceramide/sphingolipid pathway for neurodegeneration centers on glucosylceramide synthase (GCS), the enzyme that catalyzes the first step in glycosphingolipid biosynthesis: the conversion of [ceramide](/mechanisms/ceramide-signaling-neurodegeneration) to glucosylceramide[@marcaud2007].
The therapeutic rationale for GCS inhibition in neurodegeneration is strongly reinforced by the well-established association between heterozygous GBA mutations (encoding glucocerebrosidase, the enzyme that degrades glucosylceramide) and increased risk of Parkinson's disease — GBA mutations are the most common genetic risk factor for PD, present in 5-10% of all PD patients[@schapira2019][@paredes2021].
Sanofi S.A. is a French multinational pharmaceutical company headquartered in Paris, with a significant investment in rare disease therapies and lysosomal storage disorders. Sanofi's interest in the ceramide/sphingolipid pathway for neurodegeneration centers on glucosylceramide synthase (GCS), the enzyme that catalyzes the first step in glycosphingolipid biosynthesis: the conversion of [ceramide](/mechanisms/ceramide-signaling-neurodegeneration) to glucosylceramide[@marcaud2007].
The therapeutic rationale for GCS inhibition in neurodegeneration is strongly reinforced by the well-established association between heterozygous GBA mutations (encoding glucocerebrosidase, the enzyme that degrades glucosylceramide) and increased risk of Parkinson's disease — GBA mutations are the most common genetic risk factor for PD, present in 5-10% of all PD patients[@schapira2019][@paredes2021].
Sanofi's lead GCS inhibitor, eliglustat tartrate (Cerdelga), is FDA-approved for Gaucher disease type 1 and represents the most clinically advanced GCS inhibitor being repurposed for PD.