2D 3D
	ID	DDInter1218
	Drug Type	small molecule
	Molecular Formula	C10H21NO4
	Molecular Weight	219.278
	CAS Number	72599-27-0
	Description	Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.
	ATC Classification	A16AX06
IUPAC Name	(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
InChI	UQRORFVVSGFNRO-UTINFBMNSA-N
Canonical SMILES	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO
Useful Links	DrugBank ChEBI PubChem Substance KEGG Drug ChemSpider BindingDB PharmGKB Therapeutic Targets Database Wikipedia ChEMBL ZINC