MIGLUSTAT

Post-LOESM

miglustat

Amicus Therapeutics

ANDAORALCAPSULE

Approved

Mar 2025

Lifecycle

Post-LOE

Competitive Pressure

30/100

Clinical Trials

Data completeness

4/8 — Partial

MIGLUSTAT (miglustat) by Amicus Therapeutics is glucosylceramide synthase inhibitors [moa]. First approved in 2025.

Drug data last refreshed 18h ago

$0.0BPart D

Mechanism of Action

Glucosylceramide Synthase Inhibitors

Pharmacologic Class:

Glucosylceramide Synthase Inhibitor

Indications

Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.

Clinical Trials (2)

NCT04808505Phase 3Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Started Jul 2023

36 enrolled

Glycogen Storage Disease Type II Infantile Onset

NCT05174039Phase 1/2Completed

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

Started Mar 2022

6 enrolled

Batten Disease

Company

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Data Sources

Drug label: DailyMed / FDA
Clinical trials: ClinicalTrials.gov
Patent data: FDA Orange Book
Spending data: CMS Medicare

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.