Miglustat
Miglustat is an approved active ingredient.
Chemical compound
What it is used for
- Gaucher's disease
- Glycogen storage disease II
- Niemann-Pick disease
Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option. 1.1 Type 1 Gaucher Disease Miglustat is indicated as monotherapy for the treatment…
Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).
How it works
- Ceramide glucosyltransferase inhibitor
Common side effects
The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy Tremor Diarrhea and Weight Loss Reductions in Platelet Count The most common adverse reactions (incidence ≥5%) are: diarrhea, weight loss, stomach pain, gas, nausea and vomiting headache including…
Pregnancy and breastfeeding
Based on findings from animal reproduction studies, miglustat may cause fetal harm when administered to a pregnant woman.
Brand names
United States
Zavesca
European Union
Miglustat Dipharma · Veanibek (previously Miglustat Gen.Orph) · Yargesa · Zavesca
Canada
Sandoz Miglustat · Waymade-Miglustat · Zavesca
Other trade names
Miglustat gen.orph
Approval status
- US Food and Drug Administration
- prescription · since 2003
- European Medicines Agency
- authorised · since 2002
- Health Canada
- prescription · marketed
- First approval anywhere
- 2002
Codes
- ATC
- A16AX06
- RxNorm CUI
- 402316
- ChEMBL
- CHEMBL1029
- PubChem CID
- 51634
- UNII
- ADN3S497AZ
- Wikidata
- Q425911
Frequently asked questions
What is Miglustat used for?
Miglustat is used for: Gaucher's disease, Glycogen storage disease II, and Niemann-Pick disease.
What are the brand names of Miglustat?
Brand names of Miglustat include Zavesca, Miglustat Dipharma, Veanibek (previously Miglustat Gen.Orph), Yargesa, Sandoz Miglustat, Waymade-Miglustat, and Miglustat gen.orph.
Medicines in the same class
Sources and licences
- ChEMBL (CC BY-SA 3.0)
- Wikidata (CC0 1.0)
- openFDA / DailyMed (public domain)
- European Medicines Agency (reuse with acknowledgement)
- Health Canada Drug Product Database (Open Government Licence – Canada)