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Drug

Miglustat

Pronounced

"mi-GLOO-stat"

Uses

Miglustat is used in combination with another medication (cipaglucosidase alfa) to treat a certain inherited condition (Pompe disease). Cipaglucosidase alfa works by breaking down a certain substance (glycogen) that builds up in the body due to this condition. Miglustat helps cipaglucosidase alfa stay active in the blood which helps it work better.

Miglustat must be used with cipaglucosidase alfa because it does not decrease glycogen levels when used alone.

Other Uses

This section contains uses of this drug that are not listed in the approved professional labeling for the drug but that may be prescribed by your health care professional. Use this drug for a condition that is listed in this section only if it has been so prescribed by your health care professional.

Miglustat is also available in another strength and other brands for a certain rare genetic problem (Gaucher's disease). Do not take this medication with any other product that contains miglustat.

How to Use This Medication

Read the Patient Information Leaflet if available from your pharmacist before you start taking miglustat and each time you get a refill. If you have any questions, ask your doctor or pharmacist.

Take this medication by mouth on an empty stomach as directed by your doctor, usually once every other week, 1 hour before the cipaglucosidase alfa infusion. Swallow the capsules whole with only unsweetened beverages (such as water, or tea or coffee with no cream, sugar, or sweeteners). Do not drink other beverages or eat food for at least 2 hours before and 2 hours after taking this medication.

The dosage is based on your medical condition, weight, and response to treatment.

Use this medication regularly to get the most benefit from it. To help you remember, mark your calendar with a reminder. If you miss your dose of miglustat, ask your doctor or pharmacist right away for a new dosing schedule. Your cipaglucosidase alfa infusion will also need to be rescheduled.

Do not increase your dose or use this drug more often or for longer than prescribed. Your condition will not improve any faster, and your risk of side effects will increase.

Tell your doctor if your condition does not get better or if it gets worse.

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Drug information is selected from data included with permission and copyrighted by First DataBank, Inc. This is a summary and does not contain all possible information about this product. For complete information about this product or your specific health needs, ask your healthcare professional. Always seek the advice of your healthcare professional if you have any questions about this product or your medical condition. This information is not intended as individual medical advice and does not substitute for the knowledge and judgment of your healthcare professional. This information does not contain any assurances that this product is safe, effective or appropriate for you.

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Information expires December 2025.