Used in the treatment of Gaucher disease type 1 for patients who cannot receive enzyme replacement therapy. It works by reducing the production of glucosylceramide, a fatty substance that accumulates in the body due to enzyme deficiency. This helps decrease liver and spleen enlargement and improves blood cell levels. Also investigated for use in Niemann-Pick type C disease, where it may help slow neurological progression by inhibiting glycosphingolipid synthesis in the brain. Taken orally, it offers a non-injectable treatment option, allowing for easier long-term management of chronic symptoms.