Larglustat contains Eliglustat, an oral substrate reduction therapy (SRT) used for the treatment of Gaucher disease type 1 in adults. It works by reducing the production of glucosylceramide, the lipid that accumulates due to enzyme deficiency in Gaucher disease. Eliglustat is a non-enzyme therapy, unlike enzyme replacement treatments.

Mechanism of Action

• Eliglustat inhibits glucosylceramide synthase, the enzyme responsible for forming glucosylceramide.

• This reduces the formation of glycolipids that accumulate in macrophages.

• By lowering substrate buildup, it helps restore balance between lipid production and degradation, improving organ function and blood parameters.