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.
Synonyms
Butyldeoxynojirimycin
Miglustatum
1,5-(butylimino)-1,5-dideoxy, D-glucitol
Miglustat
N-butyl-deoxynojirimycin
NB-DNJ
N-(n-Butyl)deoxynojirimycin
AT2221
N-butyl-1-deoxynojirimycin
Miglustat hydrochloride
N-Butylmoranoline
n-Butyl deoxynojirimycin
BuDNJ
Brand Names
Zavesca
Opfolda
Miglustat Dipharma
Sandoz Miglustat
Yargesa
Miglustat
Miglustat Gen.orph
Indication
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).
Drug Info/Drug Targets: DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Knox C, Law V, Jewison
T, Liu P, Ly S, Frolkis A, Pon A, Banco K, Mak C, Neveu V, Djoumbou Y, Eisner R, Guo AC, Wishart DS.
Nucleic Acids Res. 2011 Jan; 39 (Database issue):D1035-41. | PMID:21059682
