About Galafold | GALAFOLD®

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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Galafold® (migalastat)

Fabry disease

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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.

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

2006

the first patient was treated in a clinical trial4

>1500

patients worldwide are being treated with Galafold as of August 20214

>90%

adherence 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

†

Data on file – August 2021. Calculated based on prescription shipment data.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

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.

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

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

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