Migalastat
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Also known as At-1001gr-181314aGalafoldSID11114003
Summary
Migalastat (CHEMBL110458) is an approved small molecule (ATC A16AX14) targeting GAA; indicated across 1 condition including fabry disease.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A16AX14
- Targets: 1 (GAA)
- Indications: 1 condition
- Clinical trials: 9
- Chemistry: 163.17 Da · C6H13NO4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL110458 |
| Name | Migalastat |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 176077 |
| ATC | A16AX14 |
| Molecular formula | C6H13NO4 |
| Molecular weight | 163.17 |
| InChIKey | LXBIFEVIBLOUGU-DPYQTVNSSA-N |
SMILES: C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O
IUPAC name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
Also known as: At-1001, gr-181314a, Galafold, Migalastat, SID11114003, MIGALASTAT, migalastat
Parent form; salt/anhydrous children: CHEMBL2107355
Patent coverage: 139 distinct patent families (430 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| GAA | alpha glucosidase | Binding | 7.4 | 0.4% | P10253 |
Broader ChEMBL bioactivity targets: 16 (assay-derived). Sample: Lactase/phlorizin hydrolase, Beta-galactosidase, Maltase-glucoamylase, Menin/Histone-lysine N-methyltransferase MLL, Lysosomal acid glucosylceramidase, Beta-galactosidase, Alpha-galactosidase A, Lysosomal alpha-glucosidase, Sucrase-isomaltase, intestinal, Polyunsaturated fatty acid lipoxygenase ALOX15.
Bioactivity
ChEMBL activities: 15 potent at pChembl ≥ 5 of 23 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| Q42656 | 8.52 | IC50 | 3 | nM | CHEMBL_ACT_3342719 |
| GLA | 7.89 | IC50 | 13 | nM | CHEMBL_ACT_18001636 |
| Q42656 | 7.89 | Ki | 13 | nM | CHEMBL_ACT_3353523 |
| GLA | 7.38 | IC50 | 42 | nM | CHEMBL_ACT_18001643 |
| GLA | 7.16 | IC50 | 70 | nM | CHEMBL_ACT_1405782 |
| GLA | 7.16 | IC50 | 70 | nM | CHEMBL_ACT_3342720 |
| GAA | 7.08 | Potency | 82.3 | nM | CHEMBL_ACT_4898651 |
| SI | 6.58 | IC50 | 260 | nM | CHEMBL_ACT_3204035 |
| GBA1 | 5.85 | Potency | 1422 | nM | CHEMBL_ACT_4741462 |
| GBA1 | 5.85 | Potency | 1422 | nM | CHEMBL_ACT_4793382 |
| MGAM | 5.82 | IC50 | 1500 | nM | CHEMBL_ACT_3204048 |
| Q9UUZ4 | 5.75 | IC50 | 1800 | nM | CHEMBL_ACT_1405791 |
| GAA | 5.22 | IC50 | 6000 | nM | CHEMBL_ACT_3204099 |
| AGL | 5 | IC50 | 10000 | nM | CHEMBL_ACT_3204111 |
| GAA | 5 | Potency | 10000 | nM | CHEMBL_ACT_4403882 |
Target pathways
Aggregated over 1 target gene(s): GAA.
Top Reactome pathways
12 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Metabolism | 1 | GAA |
| Disease | 1 | GAA |
| Innate Immune System | 1 | GAA |
| Immune System | 1 | GAA |
| Glycogen storage diseases | 1 | GAA |
| Glycogen storage disease type II (GAA) | 1 | GAA |
| Diseases of carbohydrate metabolism | 1 | GAA |
| Diseases of metabolism | 1 | GAA |
| Neutrophil degranulation | 1 | GAA |
| Glycogen breakdown (glycogenolysis) | 1 | GAA |
| Metabolism of carbohydrates and carbohydrate derivatives | 1 | GAA |
| Glycogen metabolism | 1 | GAA |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| maltose metabolic process | 1 |
| regulation of the force of heart contraction | 1 |
| diaphragm contraction | 1 |
| heart morphogenesis | 1 |
| glycogen catabolic process | 1 |
| sucrose metabolic process | 1 |
| glucose metabolic process | 1 |
| lysosome organization | 1 |
| locomotory behavior | 1 |
| tissue development | 1 |
| aorta development | 1 |
| obsolete vacuolar sequestering | 1 |
| muscle cell cellular homeostasis | 1 |
| neuromuscular process controlling posture | 1 |
| neuromuscular process controlling balance | 1 |
Indications & clinical
Indications
1 indication (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| Fabry disease | 3 | MONDO:0010526 | MONDO:0010526 |
Clinical trials
Total trials: 9.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 6 |
| Not specified | 3 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04020055 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease |
| NCT05280548 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease |
| NCT06904261 | PHASE3 | RECRUITING | A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants |
| NCT02194985 | PHASE3 | COMPLETED | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease |
| NCT03500094 | PHASE3 | COMPLETED | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) |
| NCT04049760 | PHASE3 | COMPLETED | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease |
| NCT01476163 | Not specified | AVAILABLE | Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease |
| NCT04252066 | Not specified | RECRUITING | A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding |
| NCT03949920 | Not specified | UNKNOWN | A Study of Migalastat in Fabry Disease |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline, but PharmGKB curates 19 clinical and 20 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
Related molecules
Related molecules
Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.
108 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| ACARBOSE | ChEMBL | Phase 4 (approved) | GAA |
| ACRISORCIN | ChEMBL | Phase 4 (approved) | GAA |
| ALFUZOSIN | ChEMBL | Phase 4 (approved) | GAA |
| AMILORIDE | ChEMBL | Phase 4 (approved) | GAA |
| AMLEXANOX | ChEMBL | Phase 4 (approved) | GAA |
| APOMORPHINE | ChEMBL | Phase 4 (approved) | GAA |
| ARIPIPRAZOLE | ChEMBL | Phase 4 (approved) | GAA |
| ASTEMIZOLE | ChEMBL | Phase 4 (approved) | GAA |
| BETAXOLOL | ChEMBL | Phase 4 (approved) | GAA |
| CARBARIL | ChEMBL | Phase 4 (approved) | GAA |
| CARVEDILOL | ChEMBL | Phase 4 (approved) | GAA |
| CHLORPROMAZINE | ChEMBL | Phase 4 (approved) | GAA |
| CHLORTETRACYCLINE | ChEMBL | Phase 4 (approved) | GAA |
| CINNARIZINE | ChEMBL | Phase 4 (approved) | GAA |
| DEFEROXAMINE | ChEMBL | Phase 4 (approved) | GAA |
| DEMECLOCYCLINE | ChEMBL | Phase 4 (approved) | GAA |
| DIBUCAINE | ChEMBL | Phase 4 (approved) | GAA |
| DICLOFENAC | ChEMBL | Phase 4 (approved) | GAA |
| DIENESTROL | ChEMBL | Phase 4 (approved) | GAA |
| DIPYRIDAMOLE | ChEMBL | Phase 4 (approved) | GAA |
| DITHIAZANINE IODIDE | ChEMBL | Phase 4 (approved) | GAA |
| DOPAMINE | ChEMBL | Phase 4 (approved) | GAA |
| DOXAZOSIN | ChEMBL | Phase 4 (approved) | GAA |
| DULOXETINE | ChEMBL | Phase 4 (approved) | GAA |
| ECONAZOLE NITRATE | ChEMBL | Phase 4 (approved) | GAA |
| EDARAVONE | ChEMBL | Phase 4 (approved) | GAA |
| EPINEPHRINE BITARTRATE | ChEMBL | Phase 4 (approved) | GAA |
| FELBINAC | ChEMBL | Phase 4 (approved) | GAA |
| FLUOROMETHOLONE | ChEMBL | Phase 4 (approved) | GAA |
| FLUSPIRILENE | ChEMBL | Phase 4 (approved) | GAA |
| FOLIC ACID | ChEMBL | Phase 4 (approved) | GAA |
| INDAPAMIDE | ChEMBL | Phase 4 (approved) | GAA |
| INDOPROFEN | ChEMBL | Phase 4 (approved) | GAA |
| IRBESARTAN | ChEMBL | Phase 4 (approved) | GAA |
| ISOETHARINE | ChEMBL | Phase 4 (approved) | GAA |
| ISOPROTERENOL | ChEMBL | Phase 4 (approved) | GAA |
| LABETALOL | ChEMBL | Phase 4 (approved) | GAA |
| LANSOPRAZOLE | ChEMBL | Phase 4 (approved) | GAA |
| MAPROTILINE | ChEMBL | Phase 4 (approved) | GAA |
| MECLOFENAMATE | ChEMBL | Phase 4 (approved) | GAA |
| MENADIONE | ChEMBL | Phase 4 (approved) | GAA |
| METHYLDOPA | ChEMBL | Phase 4 (approved) | GAA |
| METHYSERGIDE | ChEMBL | Phase 4 (approved) | GAA |
| MICONAZOLE NITRATE | ChEMBL | Phase 4 (approved) | GAA |
| MIGLITOL | ChEMBL | Phase 4 (approved) | GAA |
| MIGLUSTAT | ChEMBL | Phase 4 (approved) | GAA |
| MORICIZINE | ChEMBL | Phase 4 (approved) | GAA |
| NIACIN | ChEMBL | Phase 4 (approved) | GAA |
| OLANZAPINE | ChEMBL | Phase 4 (approved) | GAA |
| OXYTETRACYCLINE | ChEMBL | Phase 4 (approved) | GAA |
| PHENELZINE | ChEMBL | Phase 4 (approved) | GAA |
| PHENYLEPHRINE | ChEMBL | Phase 4 (approved) | GAA |
| PRAZOSIN | ChEMBL | Phase 4 (approved) | GAA |
| PRILOCAINE | ChEMBL | Phase 4 (approved) | GAA |
| PROCHLORPERAZINE | ChEMBL | Phase 4 (approved) | GAA |
| QUINESTROL | ChEMBL | Phase 4 (approved) | GAA |
| RABEPRAZOLE | ChEMBL | Phase 4 (approved) | GAA |
| RILUZOLE | ChEMBL | Phase 4 (approved) | GAA |
| TERAZOSIN | ChEMBL | Phase 4 (approved) | GAA |
| TERFENADINE | ChEMBL | Phase 4 (approved) | GAA |
Related Atlas pages
- Genes: GAA
- Diseases: Fabry disease
- Drugs: Acarbose, Acrisorcin, Alfuzosin, Amiloride, Amlexanox, Apomorphine, Aripiprazole, Astemizole, Betaxolol, Carbaril, Carvedilol, Chlorpromazine, Chlortetracycline, Cinnarizine, Deferoxamine, Demeclocycline, Dibucaine, Diclofenac, Dienestrol, Dipyridamole, Dopamine, Doxazosin, Duloxetine, Econazole Nitrate, Edaravone, Felbinac, Fluorometholone, Fluspirilene, Folic Acid, Indapamide, Indoprofen, Irbesartan, Isoetharine, Isoproterenol, Labetalol, Lansoprazole, Maprotiline, Meclofenamate, Menadione, Methyldopa, Methysergide, Miconazole Nitrate, Miglitol, Miglustat, Moricizine, Niacin, Olanzapine, Oxytetracycline, Phenelzine, Phenylephrine, Prazosin, Prilocaine, Prochlorperazine, Quinestrol, Rabeprazole, Riluzole, Terazosin, Terfenadine