Last reviewed 2026-05-31 · How we verify

Galafold (MIGALASTAT)

Galafold (generic name: MIGALASTAT) is a Small molecule drug developed by Amicus Therap Us. It is currently FDA-approved (first approved 2018) for Fabry's disease.

Galafold stabilizes the alpha-galactosidase A enzyme to treat Fabry's disease.

Galafold is a small molecule drug that stabilizes the enzyme alpha-galactosidase A. It is used to treat Fabry Disease, a rare genetic disorder.

At a glance

Mechanism of action

Migalastat is pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at lower pH and at higher concentration of relevant substrates, migalastat dissociates from alpha-Gal 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 protein which, however, retain enzymatic activity. Those GLA variants, referred to as amenable variants, produce alpha-Gal proteins that may be stabilized by migalastat thereby restoring their trafficking to lysosomes and their intralysosomal ac

Approved indications

• Fabry's disease

Common side effects

• Headache
• Nasopharyngitis
• Urinary tract infection
• Nausea
• Pyrexia
• Abdominal pain
• Back pain
• Cough
• Diarrhea
• Epistaxis
• Vomiting
• Cystitis

Key clinical trials

• A Study of Patients With Fabry Disease (US Specific)
• A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (PHASE3)
• Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease (PHASE3)
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants (PHASE3)
• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (PHASE3)
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease (PHASE3)
• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
• Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Patents

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Galafold CI brief — competitive landscape report
• Galafold updates RSS · CI watch RSS
• Amicus Therap Us portfolio CI

Frequently asked questions about Galafold

Related

• Target: All drugs targeting Alpha-galactosidase A
• Manufacturer: Amicus Therap Us — full pipeline
• Therapeutic area: All drugs in Metabolic
• Indication: Drugs for Fabry's disease

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing