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: *
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: Eastern America
Company CEO: Joaquin Duato
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Canada, France, Germany, Italy, Japan, Netherlands, Taiwan, United Kingdom, United States
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Glycogen Storage Disease Type II
Phase 2: Neuronal Ceroid-Lipofuscinosis
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05174039 |
Batten-1-01 | P2 |
Completed |
Neuronal Ceroid-Lipofuscinosis |
2024-05-30 |
12% |
2025-06-14 |
Primary Completion Date|Primary Endpoints|Study Completion Date |
NCT03911505 |
ATB200-04 | P3 |
Active, not recruiting |
Glycogen Storage Disease Type II |
2026-06-01 |
25% |
2025-10-28 |
Patient Enrollment|Primary Endpoints|Treatments|Trial Status |
2022-501095-25-01 |
ATB200-08 | P3 |
Recruiting |
Glycogen Storage Disease Type II |
2028-07-01 |
2025-05-02 |
Treatments |
|
NCT04808505 |
ROSSELLA | P3 |
Recruiting |
Glycogen Storage Disease Type II |
2027-07-01 |
14% |
2026-01-23 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
2022-502547-36-00 |
ATB200-04 | P3 |
Recruiting |
Glycogen Storage Disease Type II |
2026-06-01 |
2025-05-02 |
Treatments |