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 | |
---|---|---|---|
ZAVESCA Capsule, hard | European Medicines Agency (EU) | MPI, EU: SmPC | |
OPFOLDA Hard capsule | 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. |
© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.