Cerdelga mechanism of action?

How does Cerdelga work (mechanism of action)?

Cerdelga (eliglustat) is designed to lower the level of a lipid called glucosylceramide in certain cells. It does this by inhibiting an enzyme called glucosylceramide synthase, which is involved in making glucosylceramide from its precursor substrates in the cell. By blocking that enzyme, Cerdelga reduces glucosylceramide accumulation that occurs in Gaucher disease types that are driven by glucocerebrosidase deficiency.[1]

What pathway does Cerdelga affect in Gaucher disease?

In Gaucher disease, glucosylceramide (also called glucocerebroside) builds up because the body cannot break it down properly. Cerdelga targets the synthesis side of that pathway. Instead of replacing the missing enzyme (as enzyme replacement therapies do), it reduces new glucosylceramide production by inhibiting glucosylceramide synthase.[1]

How is Cerdelga different from enzyme replacement therapy?

Cerdelga works through substrate reduction (lowering the amount of glucosylceramide made), while enzyme replacement works by supplying the missing glucocerebrosidase enzyme. Because they act at different points in the disease biology, their mechanisms are not the same.[1]

Sources

[1] https://www.drugs.com/pro/cerdelga.html