Request a visit from your Galafold® (migalastat) Rare Disease Specialist

Find out if Galafold may be an appropriate treatment for your 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.

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Important safety information AND INDICATION

IMPORTANT SAFETY INFORMATION

ADVERSE REACTIONS

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

USE IN SPECIFIC POPULATIONS

There is insufficient clinical data on Galafold use in pregnant women to inform a drug associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.

It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.

The safety and effectiveness of Galafold have not been established in pediatric patients.

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4-AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

INDICATIONS AND USAGE

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.

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.

Please see accompanying Full Prescribing Information.

Keep your Fabry patients
moving forward with Galafold

Not actual patients with Fabry disease.

Keep your Fabry patients moving forward with Galafold

For adults with confirmed Fabry disease and an amenable GLA variant

Galafold—the first oral precision medicine for 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

For adults with confirmed Fabry disease and an amenable GLA variant

Galafold—the first oral precision medicine for 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 state2, 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

98%
of patients who have taken Galafold for at least 1 year stayed on Galafold4†
More than
1300
patients worldwide are being treated with Galafold as of July 20204
The first patient was treated with Galafold in
2006
in clinical trials4

*

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

Approximately 67% of patients who have taken Galafold since approval in August 2018 stayed on Galafold for at least 1 year.4

Please see Important Safety Information for Galafold below.

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.

Help your patients with Fabry disease keep moving forward with Galafold
REQUEST A REP

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:

Help your patients with Fabry disease keep moving forward with Galafold
Help your patients with Fabry disease keep moving forward with Galafold
REQUEST A REP