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Miglustat

Alternative Names: miglustat, zavesca, butyldeoxynojirimycin, ogt 918, ogt-918, ogt918
Latest Update: 2024-09-24
Latest Update Note: News Article

Product Description

Miglustat is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease. (Sourced from: https://pubchem.ncbi.nlm.nih.gov/compound/Miglustat)

Mechanisms of Action: GCS Inhibitor

Novel Mechanism: No

Modality: Small Molecule

Route of Administration: Oral

FDA Designation: None *

Approval Status: Approved

Approved Countries: Argentina | Australia | Austria | Belgium | Brazil | Bulgaria | Canada | Chile | Colombia | Croatia | Czech | Denmark | Ecuador | Egypt | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Mexico | Netherlands | New Zealand | Norway | Pakistan | Peru | Poland | Portugal | Romania | Russia | Slovakia | Slovenia | South Africa | Spain | Sweden | Switzerland | Taiwan | Thailand | Turkey | United Kingdom | United States | Uruguay | Venezuela

Approved Indications: None

Known Adverse Events: None

Company: Johnson & Johnson
Company Location: NEW BRUNSWICK NJ 08933
Company CEO: Joaquin Duato
Additonal Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Miglustat

Countries in Clinic: France, Germany, Italy, Netherlands, United States

Active Clinical Trial Count: 4

Highest Development Phases

Phase 3: Glycogen Storage Disease Type II

Phase 2: Neuronal Ceroid-Lipofuscinosis

Trial

Phase

Trial Status

Disease

Primary Completion Date

Probability of Success

ROSSELLA

P3

Recruiting

Glycogen Storage Disease Type II

2027-04-01

ATB200-08

P3

Not yet recruiting

Glycogen Storage Disease Type II

2027-04-01

ATB200-04

P3

Not yet recruiting

Glycogen Storage Disease Type II

2026-06-01

Batten-1-01

P2

Active, not recruiting

Neuronal Ceroid-Lipofuscinosis

2024-05-15

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