Drugs / Miglustat
Miglustat
also known as
OGT918 · AT2221 · Zavesca · Butyldeoxynojirimycin · mig · Miglustat dipharma · Miglustat gen.orph · N-butyldeoxynojirimycin · +3 more
Small molecule targets CFTR, UGCG via restoration / inhibition
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Refused (EMA) | EU (EMA) | Zavesca | — | 2009-06-16 | europa.eu ↗ |
| Approved | US (FDA) | ZAVESCA | — | 2003-07-31 | fda.gov ↗ |
| Approved | EU (EMA) | Zavesca | — | 2002-11-20 | europa.eu ↗ |
Trials 27 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT01822028 | Mar → Jun 2013 | diarrheal disease | Actelion | Completed | No outcome recorded |
| Phase 1 | NCT00316498 | Oct 2002 → Aug 2007 | Niemann-Pick disease type C | National Eye Institute (NEI) | Completed | No outcome recorded |
| Phase 1 | NCT00000692 | — | HIV infectious disease | G D Searle | Completed | No outcome recorded |
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT05174039 | Mar 2022 → May 2024 | juvenile neuronal ceroid lipofuscinosis 3 | Beyond Batten Disease Foundation | Completed | No outcome recorded |
| Phase 1/2 | NCT02675465 | Apr 2016 → Aug 2024 | glycogen storage disease II | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 28 trials | ||||||
| Phase 2 | NCT04768166 | Jun → Aug 2021 | hereditary spastic paraplegia | IRCCS Fondazione Stella Maris | Completed | No outcome recorded |
| Phase 2 | NCT00742092 | Aug → Dec 2008 | cystic fibrosis | Actelion | Completed | No outcome recorded |
| Phase 2 | NCT00537602 | Nov 2007 → Feb 2008 | cystic fibrosis | Actelion | Terminated | No outcome recorded |
| Phase 2 | NCT00418847 | Jul 2004 → Apr 2009 | GM2 gangliosidosis | The Hospital for Sick Children | Completed | No outcome recorded |
| Phase 2 | NCT00041535 | Jul 2002 → Mar 2007 | Gaucher disease | National Institute of Neurological Disorders and Stroke (NINDS) | Completed | No outcome recorded |
| Phase 2 | NCT00517153 | Jan 2002 → Sep 2006 | Niemann-Pick disease type C | Actelion | Completed | No outcome recorded |
| Phase 2 | NCT00001993 | — | HIV infectious disease | G D Searle | Completed | No outcome recorded |
| Phase 2 | NCT00002079 | — | HIV infectious disease | G D Searle | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT02325362 | Mar 2015 → Apr 2017 | cystic fibrosis | Assistance Publique - Hôpitaux de Paris | Completed | No outcome recorded |
| Phase 38 trials | ||||||
| Phase 3 | NCT04808505 | Jul 2023 → Jul 2027 expected | glycogen storage disease due to acid maltase deficiency, infantile onset | Amicus Therapeutics | Recruiting | No outcome recorded |
| Phase 3 | NCT03911505 | Feb 2020 → Jun 2026 overdue | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04138277 | Dec 2019 → Dec 2024 | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT03822013 | Jan 2019 → Sep 2025 | GM2 gangliosidosis | Tehran University of Medical Sciences | Terminated | No outcome recorded |
| Phase 3 | NCT03729362 | Dec 2018 → Dec 2020 | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT01760564 | Jan 2008 → Dec 2010 | Niemann-Pick disease type C | National Taiwan University Hospital | Completed | No outcome recorded |
| Phase 3 | NCT00319046 | Feb 2006 → Jun 2010 | Gaucher disease type I | Actelion | Completed | No outcome recorded |
| Phase 3 | NCT00672022 | Jul 2004 → Aug 2007 | Sandhoff disease, Tay-Sachs disease | Children's National Research Institute | Completed | No outcome recorded |
| Phase 43 trials | ||||||
| Phase 4 | NCT03910621 | Apr 2020 → Mar 2022 | Niemann-Pick disease type C | Actelion | Completed | No outcome recorded |
| Phase 4 | NCT02030015 | Dec 2015 → Jul 2019 | GM1 gangliosidosis, Sandhoff disease, Tay-Sachs disease | University of Minnesota | Terminated | No outcome recorded |
| Phase 4 | NCT00194649 | Jun 2005 → Jan 2006 | — | University of Washington | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT02520934 | Jul 2015 → Dec 2017 | Gaucher disease | National Taiwan University Hospital | Unknown | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT03865836 | — | glycogen storage disease II | Amicus Therapeutics | Available | No outcome recorded |
Also used as a comparator or background therapy in 3 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02185651 Comparator | Oct 2016 → Jul 2018 | glycogen storage disease II, hypersensitivity reaction disease | University of Florida | Terminated | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT00945347 Comparator | Jul 2009 → May 2011 | cystic fibrosis | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT04327973 Background | — | glycogen storage disease due to acid maltase deficiency, infantile onset | Amicus Therapeutics | Available | No outcome recorded |
Evidence & citations 17 cited values
Every value below carries the sentence it was read from. 32 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Miglustat | “Participants received ATB200 co-administered with AT2221 capsule (Miglustat)” NCT04327973 ↗20“Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221) capsule” NCT03911505 ↗ “Zavesca® (Miglustat) in Healthy Subjects” NCT01822028 ↗ |
| Known as | AT2221 | “Participants received ATB200 co-administered with AT2221 capsule (Miglustat)” NCT04327973 ↗ |
| Known as | Butyldeoxynojirimycin | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00001993 ↗ |
| Known as | mig | “Cipa is used with miglustat (mig), an oral enzyme stabilizer that protects cipa against breakdown in the bloodstream and increases the amount available for uptake into muscle cells.” PMID 41631150 ↗ Jan 2026 |
| Known as | Miglustat dipharma | ChEMBL registry synonym — accepted as the source's own label CHEMBL1029 ↗ |
| Known as | Miglustat gen.orph | ChEMBL registry synonym — accepted as the source's own label CHEMBL1029 ↗ |
| Known as | N-butyldeoxynojirimycin | ChEMBL registry synonym — accepted as the source's own label CHEMBL1029 ↗ |
| Known as | OGT918 | “The primary objective of the study is to investigate the pharmacokinetics of ZAVESCA® (miglustat, OGT918), when given as a single dose and at steady state, in infantile...” NCT00672022 ↗4“The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat, OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.” NCT00418847 ↗ “a new drug called OGT 918” NCT00316498 ↗ |
| Known as | Opfolda | ChEMBL registry synonym — accepted as the source's own label CHEMBL1029 ↗ |
| Known as | SC-48334 | “Phase II Study of the Safety and Surrogate Marker Efficacy of Butyldeoxynojirimycin (SC-48334) and AZT in Symptomatic HIV-1 Infected Patients With 200 - 500 CD4+ Cells/mm3.” NCT00002079 ↗ |
| Known as | Yargesa | ChEMBL registry synonym — accepted as the source's own label CHEMBL1029 ↗ |
| Known as | Zavesca | “A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects” NCT03910621 ↗ |
| Action | Inhibit | “OGT 918 is a reversible inhibitor of glucosylceramide synthase, a key enzyme in the synthesis of glycolipids” NCT00041535 ↗1“Ceramide glucosyltransferase inhibitor” CHEMBL1029 ↗ |
| Modality | Small molecule | “This inhibitor was Miglustat, which is an alkylated imino sugar.” NCT00194649 ↗ |
| Route | Oral | “the drug is administered by mouth” NCT00000692 ↗ |
| Target | CFTR | “Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR)” NCT02325362 ↗ |
| Target | UGCG | “Ceramide glucosyltransferase inhibitor” CHEMBL1029 ↗ |