Migalastat

Migalastat works by attaching to and stabilizing an enzyme in the body known as alpha-galactosidase A. A lack of this enzyme leads to 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 showed a positive response to migalastat, but more research is needed.

Migalastat may also be used for reasons not mentioned in this medication guide.

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.

Follow all instructions on your medicine label and package. Inform each of your healthcare providers about all your medical conditions, allergies, and all medicines you are taking.

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.

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

Call your doctor if you experience:

• 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 happen. Contact your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

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.

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, drinks, or activities.

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.

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