Migalastat

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.

• GALAFOLD is indicated 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 [see Dosage and Administration (2.1) and Clinical Pharmacology (12.1) ] . This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate [see Clinical Studies (14) ] .

• None. None.contraindicated

Select adults with confirmed Fabry disease who have an amenable GLA variant for treatment with GALAFOLD. ( 2.1 ) Treatment is indicated for patients with an amenable GLA variant that is interpreted by a clinical genetics professional as causing Fabry disease (pathogenic, likely pathogenic) in the clinical context of the patient. Consultation with a clinical genetics professional is strongly recommended in cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease). ( 2.1 , 12.1 ) The recommended dosage of GALAFOLD is 123 mg orally once every other day. Take GALAFOLD at the same time of day and do not take on consecutive days. Swallow capsule whole. Do not cut, crush, or chew the capsule. ( 2.2 ) Take GALAFOLD on an empty stomach. Do not consume food or caffeine at least 2 hours prior to and 2 hours after taking GALAFOLD to give a minimum 4 hour fast. ( 2.2 ) If the GALAFOLD dose is missed, take the missed dose if it is within 12 hours of the time that the dose should have been taken. If more than 12 hours have passed, take GALAFOLD at the next planned dosing day and time following the original every-other-day dosing schedule. ( 2.3 ) 2.1 Patient Selection Select adults with confirmed Fabry disease who have an amenable GLA variant for treatment with GALAFOLD [see Clinical Pharmacology (12.1) ] .

Most common adverse drug reactions ≥ 10% are: headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In clinical trials, 139 patients with Fabry disease (79 females, 60 males, 92% Caucasian, ages 16 to 72 years), who were naïve to GALAFOLD or previously treated with enzyme replacement therapy, were exposed to at least one dose of GALAFOLD. Of the 139 patients, 127 patients were exposed to GALAFOLD 123 mg every other day for 6 months and 123 patients were exposed for greater than one year. The clinical trials included one randomized, double-blind, placebo-controlled clinical trial of 6 months duration followed by a 6-month open-label treatment phase (Study 1) [see Clinical Studies (14) ] . A second trial was a randomized, open-label, active-controlled clinical trial of 18 months duration in patients with Fabry disease receiving enzyme replacement therapy who were randomized to either switch to GALAFOLD or continue enzyme replacement therapy (Study 2; NCT01218659). In addition, there were two open-label, long-term extension trials.

8.1 Pregnancy Risk Summary There were three pregnant women with Fabry disease exposed to GALAFOLD in clinical trials. As such, the available data are not sufficient to assess drug associated risks of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies, no adverse developmental effects were observed (see Data ) . The background risk for major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. In the U.S. general population, the background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. There is a study that collects data on pregnant women with Fabry disease, either exposed or unexposed to GALAFOLD. Healthcare providers are encouraged to register patients or obtain additional information by contacting the Pregnancy Coordinating Center at 1-888-239-0758, emailing fabrypregnancy@ubc.com, or visiting www.fabrypregnancyregistry.com. Data Animal Data No adverse developmental effects were observed with oral administration of migalastat to pregnant rats and rabbits during organogenesis at doses up to 26 and 54 times, respectively, the recommended dose based on AUC.

Metabolism

Absorption Following a single GALAFOLD oral dose of 123 mg, the absolute bioavailability (AUC) of migalastat was approximately 75% and the time to peak plasma concentration (t max ) was approximately 3 hours. Plasma migalastat exposure (AUC 0‑∞ and C max ) demonstrated dose‑proportional increases at oral doses from 75 mg to 1250 mg (doses from 0.5 to 8.3‑fold of the approved recommended dosage).

• Aug 10, 2018NDANDA208623Amicus Therap Us

1. 1Product Dose Omission Issue417.8%
2. 2Renal Impairment397.4%
3. 3Death315.9%
4. 4Off Label Use305.7%
5. 5Cerebrovascular Accident275.1%
6. 6Fatigue275.1%
7. 7Headache264.9%
8. 8Nausea224.2%
9. 9Pain224.2%
10. 10Dyspnoea214.0%
11. 11Drug Ineffective193.6%
12. 12Covid-19173.2%
13. 13Diarrhoea173.2%
14. 14Dizziness173.2%
15. 15Inappropriate Schedule Of Product Administration173.2%

The 10 most recently updated of 37 ClinicalTrials.gov registrations naming Migalastat as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study of Patients With Fabry Disease (US Specific)
  Recruiting · Observational · 450 enrolled · Amicus Therapeutics
  NCT06906367updated 2026-05-18
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
  Recruiting · Phase 3 · Interventional · 8 enrolled · Amicus Therapeutics
  NCT06904261updated 2026-05-15
• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
  Active not recruiting · Phase 3 · Interventional · 14 enrolled · Amicus Therapeutics
  NCT04020055updated 2026-04-03
• A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
  Active not recruiting · Phase 3 · Interventional · 104 enrolled · Sanofi
  NCT05280548updated 2026-03-10
• Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
  Completed · Phase 3 · Interventional · 16 enrolled · Amicus Therapeutics
  NCT04049760updated 2026-02-05
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
  Active not recruiting · Phase 3 · Interventional · 107 enrolled · Idorsia Pharmaceuticals Ltd.
  NCT03737214updated 2025-10-21
• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
  Recruiting · Observational · 20 enrolled · Amicus Therapeutics
  NCT04252066updated 2025-07-08
• Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
  Available · Expanded access · Amicus Therapeutics
  NCT01476163updated 2025-07-03
• A Study to Assess the Safety, Tolerability and Pharmacokinetics of Lucerastat (CDP923) After Multiple Dosing in Healthy Subjects
  Completed · Phase 1 · Interventional · 37 enrolled · Idorsia Pharmaceuticals Ltd.
  NCT02944474updated 2025-05-02
• A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects
  Completed · Phase 1 · Interventional · 39 enrolled · Idorsia Pharmaceuticals Ltd.
  NCT02944487updated 2025-05-01

Frequently asked questions

How does Migalastat work?

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.

What is Migalastat used for?

According to FDA labeling, Migalastat carries indications including: GALAFOLD is indicated 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 [see Dosage and Administration (2.1) and Clinical Pharmacology (12.1) ] . This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate [see Clinical Studies (14) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Migalastat?

Migalastat is classified as Various alimentary tract and metabolism products, Enzyme Interactions, Lipid Metabolism Alteration.

What are the brand names for Migalastat?

Migalastat is marketed under brand names including Galafold.

What are the contraindications for Migalastat?

Migalastat labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.