Chemical formula: C₁₀H₂₁NO₄ Molecular mass: 219.278 g/mol PubChem compound: 51634
Miglustat is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycolipids, and a pharmacokinetic enzyme stabiliser of cipaglucosidase alfa. Miglustat is indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease and for the treatment of progressive neurological manifestations in patients with Niemann-Pick type C disease. Also, miglustat must be used in combination with cipaglucosidase alfa for long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC code | Group title | Classification |
|---|---|---|
| A16AX06 | Miglustat | A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AX Various alimentary tract and metabolism products |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Title | Information Source | Document Type | |
|---|---|---|---|
| OPFOLDA Hard capsule | European Medicines Agency (EU) | MPI, EU: SmPC | |
| ZAVESCA Capsule, hard | European Medicines Agency (EU) | MPI, EU: SmPC |
Miglustat is an active ingredient of these brands:
United States (US)Brazil (BR)Canada (CA)Ecuador (EC)Finland (FI)Israel (IL)Japan (JP)Netherlands (NL)New Zealand (NZ)Turkey (TR)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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