migalastat
Other / unclassified targets GLA via restoration
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | US (FDA) | GALAFOLD | — | 2018-08-10 | fda.gov ↗ |
| Approved | EU (EMA) | Galafold | — | 2016-05-25 | europa.eu ↗ |
Trials 19
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 14 trials | ||||||
| Phase 1 | NCT02082327 | Mar → Jun 2014 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT01489995 | Oct → Dec 2011 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT01853852 | Sep → Dec 2011 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT01730482 | Aug → Sep 2011 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 26 trials | ||||||
| Phase 2 | NCT01196871 | Feb 2011 → Oct 2012 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00526071 | Sep 2007 → Sep 2012 | Fabry disease | Amicus Therapeutics | Terminated | No outcome recorded |
| Phase 2 | NCT00304512 | Sep 2006 → May 2008 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00283959 | Jun 2006 → May 2008 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00283933 | May 2006 → Mar 2008 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 2 | NCT00214500 | Jan 2006 → Jan 2008 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 38 trials · 2 missed | ||||||
| Phase 3 | NCT06904261 | Jan 2026 → Dec 2027 expected | Fabry disease | Amicus Therapeutics | Recruiting | No outcome recorded |
| Phase 3 | NCT04020055 | Oct 2022 → Dec 2026 expected | Fabry disease | Amicus Therapeutics | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04049760 | Oct 2019 → Nov 2024 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT03500094 | Sep 2018 → Feb 2021 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT02194985 | Mar 2015 → Oct 2019 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT01458119 | Oct 2011 → Feb 2016 | Fabry disease | Amicus Therapeutics | Terminated | Missed primary |
| Phase 3 | NCT01218659 | Sep 2011 → May 2014 | Fabry disease | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT00925301 | Oct 2009 → Jun 2012 | Fabry disease | Amicus Therapeutics | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT01476163 | — | Fabry disease | Amicus Therapeutics | Available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT05280548 Comparator | May 2022 → May 2026 overdue | Fabry disease | Sanofi | Active not recruiting | No outcome recorded |
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 23 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | migalastat | “Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects.” PMID 27536442 ↗ Jul 201319“A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)” NCT01730482 ↗ “migalastat” NCT04049760 ↗ |
| Known as | 1-Deoxygalactonojirimycin | “Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological chaperone for the treatment of α-galactosidase A (α-Gal A) deficiency” PMID 26252393 ↗ Aug 2015 |
| Known as | AT1001 | “This study is designed to describe the metabolism of AT1001 (migalastat HCl) and the contribution of metabolism and urinary excretion to its overall elimination as part of the...” NCT01730482 ↗ |
| Known as | AT1001, migalastat | “AT1001, migalastat” NCT01476163 ↗ |
| Known as | GR181413A/AT1001 | “Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects.” PMID 27536442 ↗ Jul 2013 |
| Known as | migalastat HCl | “The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat...” NCT02082327 ↗7“This study is designed to describe the metabolism of AT1001 (migalastat HCl) and the contribution of metabolism and urinary excretion to its overall elimination as part of the...” NCT01730482 ↗ “Each individual GLA mutation was retrospectively categorized as being amenable or not to migalastat HCl based on an in vitro α-Gal A transfection assay developed in human...” PMID 23474038 ↗ Jan 2013 “migalastat HCl 150 mg capsule” NCT03500094 ↗ “migalastat HCl 150 mg capsule” NCT04049760 ↗ “migalastat HCl 150 mg” NCT01476163 ↗ “Drug: migalastat HCl” NCT00526071 ↗ |
| Known as | migalastat hydrochloride | “One migalastat 123 milligrams (mg) capsule equivalent to 150 mg migalastat hydrochloride (HCl) (herein referred to as "migalastat") was administered every other day for 12 months.” NCT03500094 ↗5“We report on the safety and pharmacodynamics of migalastat hydrochloride, an investigational pharmacological chaperone given orally every other day (QOD) to females with FD.” PMID 23474038 ↗ Jan 2013 “Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects.” PMID 27536442 ↗ Jul 2013 “A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)” NCT01730482 ↗ “Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease” NCT00925301 ↗ “Drug: migalastat hydrochloride” NCT01458119 ↗ |
| Action | Restore | “Migalastat, a pharmacological chaperone, binds to specific mutant forms of α-galactosidase A to restore lysosomal activity.” PMID 27657681 ↗ Sep 2016 |
| Modality | Other / unclassified | “Migalastat, a pharmacological chaperone, binds to specific mutant forms of α-galactosidase A to restore lysosomal activity.” PMID 27657681 ↗ Sep 2016 |
| Route | Oral | “Migalastat was administered orally during the 12-week treatment period” NCT00214500 ↗ |
| Target | GLA | “Each individual GLA mutation was retrospectively categorized as being amenable or not to migalastat HCl based on an in vitro α-Gal A transfection assay developed in human...” PMID 23474038 ↗ Jan 2013 |