Common name

IUPAC name

(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

SMILES

CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO

Compound class

Enzyme Inhibitors; Alimentary Tract and Metabolism; Various Alimentary Tract and Metabolism Products;

Therapeutic area

For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C).