Miglustat

Chemical formula: C₁₀H₂₁NO₄  Molecular mass: 219.278 g/mol  PubChem compound: 51634

Active ingredient description

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).

Medicine classification

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
Discover more medicines within A16AX06

Product monographs

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

Structural formula

Graphic representation of the active ingredient's molecular structure

External identifiers

CAS Substance: 72599-27-0
DrugBank Drug: DB00419
KEGG Drug: D05032
PubChem Compound: 51634
RxNorm Ingredient: 402316
SNOMED-CT Concept: 412296005
Miglustat (substance)
UNII Identifier: ADN3S497AZ
MIGLUSTAT

Medicines

Miglustat is an active ingredient of these brands:

United States (US)

Austria (AT)

Brazil (BR)

Canada (CA)

Croatia (HR)

Ecuador (EC)

Estonia (EE)

Finland (FI)

France (FR)

Ireland (IE)

Israel (IL)

Italy (IT)

Japan (JP)

Lithuania (LT)

Netherlands (NL)

New Zealand (NZ)

Poland (PL)

Spain (ES)

Turkey (TR)

United Kingdom (UK)

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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