Migalastat is an alpha-galactosidase A chaperone used for the treatment of Fabry disease in patients with an amenable galactosidase alpha gene (GLA) variant.

Brand Names
Generic Name
DrugBank Accession Number

Fabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other organs.5,6,3,4 In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated.3

Migalastat (approved and sold under Amicus Therapeutics' brand name Galafold) is subsequently an oral pharmacological chaperone of alpha-Gal A for the treatment of Fabry disease in adults who have amenable GLA variants.5,6,3,4 In these patients, migalastat works by stabilizing the body’s dysfunctional alpha-Gal A enzyme so that it can clear the accumulation of glycosphingolipid disease substrate.5,6,3,4 Globally, it is estimated that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants that are treatable with migalastat. 3

Given the rarity of Fabry disease and the proportion of Fabry disease patients that could benefit from migalastat therapy, Amicus Therapeutics' brand name Galafold was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is an unmet medical need and where a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients.5,6,3,4 A further study is required to verify and describe the clinical benefits of Galafold, and the sponsor will be conducting a confirmatory clinical trial of Galafold in adults with Fabry disease.5,6,3,4

Additionally, Galafold was also granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months of application filing where the agency determines that the drug if approved, would provide a significant improvement in treating, diagnosing or preventing a serious condition over available therapies.5,6 Galafold also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.5,6

As of August 2018, migalastat under Amicus Therapeutics' brand name Galafold is currently approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland, and the United States.3

Small Molecule
Approved, Investigational
Average: 163.1717
Monoisotopic: 163.084457909
Chemical Formula
  • 1-Deoxygalactonojirimycin
  • 1-Deoxygalactostatin
  • Migalastat



Migalastat is approved by the FDA for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.5

This indication is approved under accelerated approval based on a reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.5

Migalastat is also approved by the EMA and Health Canada to treat the same disease, although it is approved for both adults and adolescents aged 16 years and older in Europe.6,7

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Associated Conditions
Indication TypeIndicationCombined Product DetailsApproval LevelAge GroupPatient CharacteristicsDose Form
Treatment ofFabry disease•••••••••••••••••••••••••••••••••••• ••••••• •••••••••••••••
Treatment ofFabry disease•••••••••••••••••••••••••••••••••••• ••••••• •••••••••••••••
Treatment ofFabry disease••••••••••••••••••••••• •••••••••••••••••••••••• ••••••• •••••••••••••••
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In general, treatment in patients with migalastat in Phase 2 pharmacodynamic trials resulted in increases in endogenous alpha-galactosidase (alpha-Gal A) activity in white blood cells, as well as in skin and kidney for the majority of patients Label. In patients with amenable galactosidase alpha gene (GLA) mutations, globotriaosylceramide (GL-3) levels tended to decrease in the urine and in the kidney interstitial capillaries.6

In an in vitro assay (HEK-293 assay), Human Embryonic Kidney (HEK-293) cell lines were transfected with specific GLA variants (mutations) which produced mutant alpha-Gal A proteins. In the transfected cells, amenability of the GLA variants was assessed after a 5-day incubation with 10 micromol/L migalastat. A GLA variant was categorized as amenable if the resultant mutant alpha-Gal A activity (measured in the cell lysates) met two criteria: 1) it showed a relative increase of at least 20% compared to the pre-treatment alpha-Gal A activity, and 2) it showed an absolute increase of at least 3% of the wild-type (normal) alpha-Gal activity. The in vitro assay did not evaluate the trafficking of the mutant alpha-Gal A proteins into the lysosome or the dissociation of migalastat from the mutant alpha-Gal A proteins within the lysosome. Also, the in vitro assay did not test whether a GLA variant causes Fabry disease or not.5

Mechanism of action

Fabry disease is a progressive X-linked lysosomal storage disorder that affects males and females Label. Fabry disease-causing mutations occur in the galactosidase alpha (GLA) gene and result in a deficiency of the lysosomal enzyme alpha-galactosidase A (alpha-Gal A) that is required for glycosphingolipid substrate (GL-3 and lyso-Gb3) metabolism Label. Reduced alpha-Gal A activity is, therefore, associated with the progressive accumulation of glycosphingolipid substrate in vulnerable organs and tissues, which ultimately leads to the morbidity and mortality associated with Fabry disease.6

Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH and at a higher concentration of relevant substrates, migalastat dissociates from alpha-Gal A allowing it to break down the glycosphingolipids globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). Certain GLA variants (mutations) causing Fabry disease result in the production of abnormally folded and less stable forms of the alpha-Gal A protein which, however, retain enzymatic activity. Those GLA variants, referred to as amenable variants, produce alpha-Gal A proteins that may be stabilized by migalastat thereby restoring their trafficking to lysosomes and their intralysosomal activity.5

The GLA mutations that are amenable and not amenable to treatment with migalastat are regularly maintained and updated on online sites that are readily accessible by healthcare providers.5

AAlpha-galactosidase A

With absorption occurring largely in the gut, the absolute bioavailability (AUC) for a single oral 150 mg migalastat hydrochloride dose or a single 2-hour 150 mg intravenous infusion was approximately 75% and Tmax was approximately 3 hours.2,6 Plasma migalastat exposure (AUC0-∞) and Cmax demonstrated dose-proportional increases at migalastat hydrochloride oral doses from 50 mg to 1,250 mg (doses from 0.5 to 8.3-fold of the approved recommended dosage).5,6

Migalastat administered with a high-fat meal (850 calories; 56% from fat), or 1 hour before a high-fat or light meal (507 calories; 30% from fat), or 1 hour after a light meal, resulted in significant reductions of 37% to 42% in mean total migalastat exposure (AUC0-∞) and reductions of 15% to 39% in mean peak migalastat exposure (Cmax) compared with the fasting state.5

Volume of distribution

In healthy volunteers, the volume of distribution (Vz/F) of migalastat following ascending single oral doses (25-675 mg migalastat HCl) ranged from 77 to 133 L, indicating it is well distributed into tissues and greater than total body water (42 liters).6

Protein binding

There was no detectable plasma protein binding following administration of [14C]-migalastat hydrochloride in the concentration range between 1 and 100 µM.5,6


Based upon in vivo data, migalastat is a substrate for uridine diphosphate glucuronosyltransferase (otherwise known as UGT or UDPGT), being a minor elimination pathway.5

Route of elimination

In a mass balance study in healthy male subjects, following oral administration of 123 mg [14C]-migalastat, approximately 77% of the total radiolabeled dose was recovered in urine and 20% of the total radiolabeled dose was recovered in feces with an overall total recovery of 98% within 96 hours post-dose. In urine, unchanged migalastat accounted for 80% of the radioactivity, which equates to 62% of the administered dose. In feces, unchanged migalastat was the only drug-related component. In plasma, unchanged migalastat accounted for approximately 77% of the plasma radioactivity, and three dehydrogenated O-glucuronide conjugated metabolites, M1 to M3, together accounted for approximately 13% of the plasma radioactivity, none of which comprised more than 6% of the radiolabeled dose. Approximately 9% of the total radioactivity in plasma was unassigned.5


The mean elimination half-life (t1/2) of migalastat ranges from approximately 3 to 5 hours for a single oral dose of 150 mg.6 For the dose of 123 mg, the mean elimination half-life was estimated to be 4 hours.5


Following ascending single oral doses (25-675 mg migalastat hydrochloride), no trends were found for clearance (CL/F). At the 150 mg dose, CL/F was approximately 11 to 14 L/hr, while at 123 mg, the apparent clearance was calculated to be 12.5 L/hr.6,5

Adverse Effects
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The most common adverse reactions reported with migalastat (≥ 10%) during the 6-month placebo-controlled, double-blind phase of its Study 1 clinical studies were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia Label.

In case of overdose, general medical care is recommended Label. Headache and dizziness were the most common adverse reactions reported at doses of migalastat of up to 1250 mg and 2000 mg, respectively Label.

Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
AbacavirAbacavir may decrease the excretion rate of Migalastat which could result in a higher serum level.
AceclofenacAceclofenac may decrease the excretion rate of Migalastat which could result in a higher serum level.
AcemetacinAcemetacin may decrease the excretion rate of Migalastat which could result in a higher serum level.
AcetaminophenAcetaminophen may decrease the excretion rate of Migalastat which could result in a higher serum level.
AcetazolamideAcetazolamide may increase the excretion rate of Migalastat which could result in a lower serum level and potentially a reduction in efficacy.
Food Interactions
  • Avoid caffeine. Co-administration of GALAFOLD with caffeine decreases migalastat AUC and Cmax which may reduce its efficacy.
  • Take at the same time every day.
  • Take on an empty stomach. Avoid eating for at least two hours before and after taking migalastat.


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Product Ingredients
IngredientUNIICASInChI Key
Migalastat hydrochlorideCLY7M0XD2075172-81-5ZJIHMALTJRDNQI-OLALXQGDSA-N
International/Other Brands
Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
GalafoldCapsule123 mgOralAmicus Therapeutics Canada Inc.2018-01-15Not applicableCanada flag
GalafoldCapsule123 mg/1OralAmicus Therapeutics US, LLC2018-08-10Not applicableUS flag
GalafoldCapsule123 mgOralAmicus Therapeutics Europe Limited2020-12-22Not applicableEU flag


ATC Codes
A16AX14 — Migalastat
Drug Categories
Chemical TaxonomyProvided by Classyfire
This compound belongs to the class of organic compounds known as piperidines. These are compounds containing a piperidine ring, which is a saturated aliphatic six-member ring with one nitrogen atom and five carbon atoms.
Organic compounds
Super Class
Organoheterocyclic compounds
Sub Class
Not Available
Direct Parent
Alternative Parents
Secondary alcohols / 1,2-aminoalcohols / Polyols / Dialkylamines / Azacyclic compounds / Primary alcohols / Organopnictogen compounds / Hydrocarbon derivatives
1,2-aminoalcohol / Alcohol / Aliphatic heteromonocyclic compound / Amine / Azacycle / Hydrocarbon derivative / Organic nitrogen compound / Organic oxygen compound / Organonitrogen compound / Organooxygen compound
Molecular Framework
Aliphatic heteromonocyclic compounds
External Descriptors
Not Available
Affected organisms
  • Humans and other mammals

Chemical Identifiers

CAS number
InChI Key


Synthesis Reference

Patent US20180208955A1: Process for the microbial synthesis of migalastat (https://patents.google.com/patent/US20180208955A1/en)

General References
  1. Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK: Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015. [Article]
  2. Migalastat for the treatment of Fabry Disease: Sunder-Plassmann G., Schiffmann R., and Nicholls K. [Link]
  3. Amicus Therapeutics News Release: FDA Approves Galafold™ (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease [Link]
  4. Drugs.com: FDA Approves Galafold (migalastat) for the Treatment of Fabry Disease [Link]
  5. FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]
  6. EMA Approved Drug Products: GALAFOLD (Migalastat) capsules for oral use [Link]
  7. Health Canada Approved Drug Proucts: GALAFOLD (Migalastat) capsules for oral use [Link]
  8. Patent US20180208955A1: Process for the microbial synthesis of migalastat [File]
PubChem Compound
PubChem Substance
PDBe Ligand
PDB Entries
3s5y / 3thd / 3tv8 / 4cu8 / 4d1j / 4do5 / 4fns / 4ufm / 6euh / 6tsh
show 1 more
FDA label
Download (1.3 MB)
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Clinical Trials

Clinical Trials
3Active Not RecruitingTreatmentFabry's Disease1
3CompletedTreatmentFabry's Disease4
3RecruitingTreatmentFabry's Disease2
3TerminatedTreatmentFabry's Disease1
2CompletedTreatmentFabry's Disease5


Not Available
Not Available
Dosage Forms
CapsuleOral123 mg
CapsuleOral123 mg/1
Capsule, coatedOral150 mg
Not Available
Patent NumberPediatric ExtensionApprovedExpires (estimated)Region
US9987263No2018-06-052027-05-16US flag
US9000011No2015-04-072027-05-16US flag
US8592362No2013-11-262029-02-12US flag
US9095584No2015-08-042029-02-12US flag
US9480682No2016-11-012027-05-16US flag
US9999618No2018-06-192028-04-28US flag
US10076514No2018-09-182037-03-15US flag
US10251873No2019-04-092038-05-30US flag
US10383864No2019-08-202027-05-16US flag
US10471053No2019-11-122038-05-30US flag
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US10525045No2020-01-072028-04-28US flag
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US10813921No2020-10-272029-02-12US flag
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US10806727No2020-10-202038-05-30US flag
US10925866No2021-02-232028-04-28US flag
US11033538No2021-06-152028-04-28US flag
USRE48608No2021-06-292029-02-12US flag
US11241422No2007-05-162027-05-16US flag
US11234972No2017-03-152037-03-15US flag
US11278538No2018-05-302038-05-30US flag
US11278537No2018-05-302038-05-30US flag
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US11278540No2018-05-302038-05-30US flag
US11304940No2018-05-302038-05-30US flag
US11357761No2008-05-302028-05-30US flag
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US11622962No2019-03-172039-03-17US flag
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US11612594No2018-05-302038-05-30US flag
US11666564No2018-05-302038-05-30US flag
US11786516No2018-05-302038-05-30US flag
US11833164No2022-01-112042-01-11US flag
US11813255No2018-05-302038-05-30US flag
US11826360No2019-02-162039-02-16US flag


Experimental Properties
Not Available
Predicted Properties
Water Solubility511.0 mg/mLALOGPS
pKa (Strongest Acidic)12.91Chemaxon
pKa (Strongest Basic)8.06Chemaxon
Physiological Charge1Chemaxon
Hydrogen Acceptor Count5Chemaxon
Hydrogen Donor Count5Chemaxon
Polar Surface Area92.95 Å2Chemaxon
Rotatable Bond Count1Chemaxon
Refractivity36.57 m3·mol-1Chemaxon
Polarizability15.86 Å3Chemaxon
Number of Rings1Chemaxon
Rule of FiveYesChemaxon
Ghose FilterNoChemaxon
Veber's RuleNoChemaxon
MDDR-like RuleNoChemaxon
Predicted ADMET Features
Not Available


Mass Spec (NIST)
Not Available
SpectrumSpectrum TypeSplash Key
Predicted GC-MS Spectrum - GC-MSPredicted GC-MSsplash10-0feb-7900000000-536465f5fd772045b134
Predicted MS/MS Spectrum - 10V, Positive (Annotated)Predicted LC-MS/MSsplash10-002b-0900000000-179be3503509a3b85d39
Predicted MS/MS Spectrum - 10V, Negative (Annotated)Predicted LC-MS/MSsplash10-01q9-1900000000-158c07bd46d97cf5da76
Predicted MS/MS Spectrum - 20V, Positive (Annotated)Predicted LC-MS/MSsplash10-004j-1900000000-54d3fe7d55f96a10c7ae
Predicted MS/MS Spectrum - 20V, Negative (Annotated)Predicted LC-MS/MSsplash10-03e9-3900000000-209067245351329bb22d
Predicted MS/MS Spectrum - 40V, Positive (Annotated)Predicted LC-MS/MSsplash10-0pbc-9400000000-030cbb5d966f8ee8d6ca
Predicted MS/MS Spectrum - 40V, Negative (Annotated)Predicted LC-MS/MSsplash10-0006-9100000000-de6e9c082c4064e26585
Predicted 1H NMR Spectrum1D NMRNot Applicable
Predicted 13C NMR Spectrum1D NMRNot Applicable
Chromatographic Properties
Collision Cross Sections (CCS)
AdductCCS Value (Å2)Source typeSource
DeepCCS 1.0 (2019)
DeepCCS 1.0 (2019)
DeepCCS 1.0 (2019)


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Pharmacological action
General Function
Receptor binding
Specific Function
Not Available
Gene Name
Uniprot ID
Uniprot Name
Alpha-galactosidase A
Molecular Weight
48766.34 Da
  1. FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]


Pharmacological action
Curator comments
Low affinity
General Function
Glucose:sodium symporter activity
Specific Function
Actively transports glucose into cells by Na(+) cotransport with a Na(+) to glucose coupling ratio of 2:1. Efficient substrate transport in mammalian kidney is provided by the concerted action of a...
Gene Name
Uniprot ID
Uniprot Name
Sodium/glucose cotransporter 1
Molecular Weight
73497.275 Da
  1. FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]

Drug created at October 21, 2007 22:23 / Updated at March 03, 2024 02:34