Drugs / Miglustat
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 24
| 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 1/21 trial | ||||||
| Phase 1/2 | NCT05174039 | Mar 2022 → May 2024 | juvenile neuronal ceroid lipofuscinosis 3 | Beyond Batten Disease Foundation | 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 | NCT00002079 | — | HIV infectious disease | G D Searle | Completed | No outcome recorded |
| Phase 2 | NCT00001993 | — | 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 37 trials · 1 met primary · 1 missed | ||||||
| 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 → Mar 2027 expected | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Active not recruiting | 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 | Missed primary |
| 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 | Met primary |
| 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 |
| Comparator or background therapy2 trials | ||||||
| Phase 1 | NCT02185651 Comparator | Oct 2016 → Jul 2018 | glycogen storage disease II, hypersensitivity reaction disease | University of Florida | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT00945347 Comparator | Jul 2009 → May 2011 | cystic fibrosis | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2026-10-02 | BioMarin Pharmaceutical | Results BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress biomarin.com ↗
These results from the PROPEL open-label extension (OLE) study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up. |
| 2026-09-01 | Theranexus | Regulatory THX Pharma, BBDF and Biocodex Announce Regulatory Submissions for the Phase 3 Clinical Trial of Batten-1 in Juvenile Batten Disease (CLN3) actusnews.com ↗
THX Pharma (Theranexus), an innovative biopharmaceutical company developing treatments for rare neurological diseases, together with its partners, the Beyond Batten Disease Foundation (BBDF) and Biocodex, today announced the submission of regulatory applications to initiate the Phase 3 clinical trial evaluating Batten-1 (study Batten-1-02), a proprietary oral liquid formulation of miglustat developed for the treatment of juvenile Batten disease (CLN3). |
| 2025-05-13 | Theranexus | Results THERANEXUS AND BEYOND BATTEN DISEASE FOUNDATION ANNOUNCE STRONG POSITIVE REAL-WORLD DATA SUPPORTING BATTEN-1 EFFICACY FOR THE TREATMENT OF BATTEN DISEASE actusnews.com ↗
The analysis demonstrated a statistically significant and clinically meaningful difference in favor of Batten-1 treatment on visual function with a notable preservation of visual acuity in treated patients compared to untreated patients. |
| 2024-11-19 | Theranexus | Results Update on the Theranexus' Batten-1 project in CLN3 Batten's disease actusnews.com ↗
This presentation highlights the strong safety profile of miglustat in CLN3 Batten disease patients and underscores the positive efficacy data gathered during this trial. |
| 2024-06-06 | Theranexus | Results Theranexus and BBDF confirm positive 18-month results for Batten-1 in Phase I/II trial based on neurofilaments, a biomarker of neuronal death actusnews.com ↗ |
| 2024-04-18 | Theranexus | Results Theranexus and BBDF announce final and positive safety and efficacy data in the Phase I/II trial testing Batten-1 actusnews.com ↗
The 18-month safety and efficacy data confirm the 12-month results announced in September 2023. |
| 2023-02-20 | Theranexus | Results THERANEXUS, BBDF AND CARDIFF UNIVERSITY PRESENT THEIR NOVEL RESEARCH ON BATTEN-1 AND BATTEN DISEASE AT WORLDSYMPOSIUM 2023 actusnews.com ↗
Presentation of preclinical data demonstrating how Batten-1 drastically reduces the buildup of toxic glycosphingolipids in Batten disease and a poster describing for the first time the burden of Batten disease. |
| 2023-01-05 | Theranexus | Results New advance to the Theranexus and BBDF Batten-1 program for juvenile Batten disease (CLN3) actusnews.com ↗
We are delighted that Batten-1 showed good safety and tolerability for our patients suffering from Batten disease. |
| 2021-09-09 | Theranexus | Regulatory THERANEXUS AND BEYOND BATTEN DISEASE FOUNDATION (BBDF) WIN US INVESTIGATIONAL NEW DRUG (IND) APPROVAL TO START CLINICAL DEVELOPMENT OF BBDF 101 actusnews.com ↗
With the FDA's IND approval for BBDF 101, we have reached a significant milestone in the drug’s regulatory development plan. |
All press releases naming this drug 11 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 27 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Miglustat | “Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221) capsule” NCT03911505 ↗19“Zavesca® (Miglustat) in Healthy Subjects” NCT01822028 ↗ |
| Known as | Butyldeoxynojirimycin | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00002079 ↗ |
| 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 | 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 | 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 | 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 ↗ |
| 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 ↗ |