The information you are about to see is intended for U.S. healthcare professionals.
Please confirm you are a U.S. healthcare professional.
I am a licensed U.S. healthcare professional
I am NOT a licensed U.S. healthcare professional
You are leaving this website
You are now leaving The site you are going to may not be owned or maintained by Amicus Therapeutics. Amicus Therapeutics is not responsible for the information contained on third-party sites.
Exit the request a representative window

Request more information or a visit from your Amicus Rare Disease Specialist

Find out if Galafold® (migalastat) may be an appropriate treatment for your adult patients with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant. Once you submit your request, a representative will contact you.

Please fill in all fields below and submit your request.

*Required fields.
Which of the following would you like more information on? (Check all that apply.)
Thank you! Your submission has been received!
Oops! Something went wrong while submitting the form.
Exit the request a representative window
Thank you for your request!
Open or close the important safety information

Galafold® (migalastat) 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.

This indication is approved under accelerated approval based on 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.

For adults with confirmed Fabry disease and an amenable GLA variant

Galafold—the first oral precision medicine for Fabry disease

Alex, living with Fabry disease

Galafold is a small-molecule alpha-Gal A pharmacological chaperone that reversibly binds to the active site of alpha-Gal A and stabilizes the protein.1,2

Galafold restores the normal pathway for trafficking amenable alpha-Gal A to the lysosome.2

Galafold has a large apparent volume of distribution of approximately 89 L (range: 77 to 133 L) at steady state,2 suggesting it is well distributed into tissues.1,3

Galafold reduced disease substrate (GL-3) in kidney interstitial capillaries (KIC) over 6 months.2*

The most common adverse drug reactions reported with Galafold (≥10%) are headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia.2

the first patient
was treated

with migalastat in a clinical trial4
patients worldwide
are being treated
with Galafold4†
for patients
on Galafold4‡
Mean change in average number of GL-3 inclusions per KIC from Baseline to Month 6 was -0.04 (95% CI: -1.94, 0.26; n=25) with Galafold treatment vs -0.03 (95% CI: -1.00, 1.69; n=20) with placebo.2
As of December 2023.4
As of December 2023. Calculated based on prescription shipment data of current patients receiving Galafold treatment.4
Please see Important Safety Information for Galafold below.

Galafold: a distinct approach to the treatment of Fabry disease

  • Pharmacological chaperones are small-molecule analogs of the protein’s substrate with the ability to bind and stabilize certain variant forms of the proteins so that they can be trafficked from the endoplasmic reticulum (ER) into the lysosome, restoring intralysosomal activity2,5-7
  • Galafold binds and stabilizes alpha-Gal A in patients with an amenable GLA variant2
  • Alpha-Gal A from an amenable GLA variant is trafficked from the ER through the Golgi apparatus to the lysosomes2

See how Galafold works

Watch this video to learn more about the mechanism of action for Galafold.

Identify whether your patient's GLA variant is amenable to treatment with Galafold

Starting with your patient's GLA genotype, you can determine whether the GLA variant is amenable in 1 of 3 ways:

The Galafold Full Prescribing Information includes a list of GLA variants that have been identified as amenable to Galafold.

Download now

Check amenability on this website to determine whether a specific GLA variant has been listed as amenable in the Full Prescribing Information.

Check now

For answers to questions about amenability, you can also contact Amicus Medical Information at 1-877-4-AMICUS or

Contact now

Start a patient

Start a patient

Download the Galafold Patient Referral Form, which serves both as a prescription for Galafold in some states and a referral to AMICUS ASSIST®.