Galafold works by binding to and stabilizing a certain enzyme in the body called alpha-galactosidase A. A deficiency of this enzyme is called Fabry disease.

Galafold is used to treat Fabry disease in adults.

Galafold was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some people responded to this medicine, but further studies are needed.

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You should not use Galafold if you are allergic to migalastat.

Tell your doctor if you have ever had kidney problems.

Tell your doctor if you are pregnant or breastfeeding.

If you are pregnant or breastfeeding, your name may be listed on a registry to track the effects of migalastat on the baby.

Galafold is not approved for use by anyone younger than 18 years old.

How should I take Galafold?

Take Galafold exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets.

Take this medicine on an empty stomach, at least 2 hours before or 2 hours after a meal.

Galafold is usually taken every other day, at the same time of day.

Swallow the capsule whole and do not crush, chew, break, or open it.

Do not take this medicine 2 days in a row.

Store at room temperature away from moisture and heat. Keep unused capsules in the blister pack.

Take Galafold exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets.

Take this medicine on an empty stomach, at least 2 hours before or 2 hours after a meal.

Galafold is usually taken every other day, at the same time of day.

Swallow the capsule whole and do not crush, chew, break, or open it.

Do not take this medicine 2 days in a row.

Store at room temperature away from moisture and heat. Keep unused capsules in the blister pack.

Usual Adult Dose for Fabry Disease:

123 mg orally once every other day
-Take on an empty stomach; do not consume food for at least 2 hours before and 2 hours after dosing; clear liquids may be consumed during this period

Comments:
-This drug should not be taken on 2 consecutive days.
-Patients with Fabry disease should have an amenable galactosidase alpha gene variant (GLA) that is interpreted by a clinical geneticist as pathogenic or likely pathogenic as causing Fabry disease.
-This drug approval is under an accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate; continued approval is subject to demonstrated clinical benefit.

Use: For the treatment of adults with Fabry disease and an amenable galactosidase alpha gene variant (GLA) based on in vitro assay data.

You should not use Galafold if you are allergic to migalastat.

Tell your doctor if you have ever had kidney problems.

Tell your doctor if you are pregnant or breastfeeding.

If you are pregnant or breastfeeding, your name may be listed on a registry to track the effects of migalastat on the baby.

Galafold is not approved for use by anyone younger than 18 years old.

Use the medicine as soon as you can, but skip the missed dose if you are more than 12 hours late for the dose. Do not use two doses at one time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction to Galafold: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor if you have:

• pain or burning when you urinate.

Common Galafold side effects may include:

• headache;

• nausea;

• fever; or

• cold symptoms such as stuffy nose, sneezing, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Other drugs may interact with migalastat, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.