Migalastat

Generic name: migalastat [ mi-GAL-a-stat ]
Brand name: Galafold
Dosage form: oral capsule (123 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Drugs.com on Dec 26, 2024. Written by Cerner Multum.

What is migalastat?

Migalastat 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.

Migalastat is used to treat Fabry disease in adults.

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

Migalastat may also be used for purposes not listed in this medication guide.

Migalastat side effects

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

Call your doctor if you have:

• pain or burning when you urinate.

Common side effects of migalastat 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.

Related/similar drugs

Warnings

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

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.

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

How should I take migalastat?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

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

Migalastat 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 migalastat 2 days in a row.

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

Migalastat dosing information

Usual Adult Dose for Fabry Disease:

123 mg orally once every other day
-Take at the same time of day 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.

What happens if I miss a dose?

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.

What happens if I overdose?

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

What should I avoid while taking migalastat?

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

What other drugs will affect migalastat?

Other drugs may affect 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.

Frequently asked questions

• How much does Galafold cost?

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer

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