Wednesday, July 8, 2026

Drug profile · INN

miglustat

miglustat (MIGLUSTAT DIPHARMA) is a ceramide glucosyltransferase inhibitor. Route of administration: oral. Current US regulatory status: approved.

FDA: approved EMA: approved Alimentary tract and metabolism (A16) A16AX06
US status approved
Patents linked 0
Pipeline programs 2
News articles 1
Drug details — miglustat

Quick answer

miglustat (MIGLUSTAT DIPHARMA) is a ceramide glucosyltransferase inhibitor. Route of administration: oral. Current US regulatory status: approved.

Key facts

INN miglustat
Brand MIGLUSTAT DIPHARMA
Mechanism Ceramide glucosyltransferase inhibitor
Target Ceramide glucosyltransferase
Therapeutic class Alimentary tract and metabolism (A16)
Route ORAL
Formulation CAPSULE
ATC code A16AX06

Mechanism of action

Ceramide glucosyltransferase inhibitor

Primary target: Ceramide glucosyltransferase

Approval history

Authority Status Date
FDA approved
EMA approved

Recent coverage

Pipeline programs

  1. Myozyme powder for concentrate for solution f... for Pompe disease Approved · Disc Medicine · Pompe disease
  2. miglustat Approved · Ultragenyx UK · GM1 Gangliosidoses

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Frequently asked questions

What is miglustat?

miglustat (MIGLUSTAT DIPHARMA) is a ceramide glucosyltransferase inhibitor. Route of administration: oral. Current US regulatory status: approved.

What is the brand name for miglustat?

MIGLUSTAT DIPHARMA is a marketed brand name for miglustat. Check FDA Drugs@FDA for the current US label holder and prescribing information.

What is the US approval status of miglustat?

miglustat has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.