Miglustat Capsules (Pompe Disease)

What is this medication?

MIGLUSTAT (MIG loo stat) treats Pompe disease. It works by increasing the effects of cipaglucosidase alfa in the body to help it work better.

This medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

COMMON BRAND NAME(S): OPFOLDA

What should I tell my care team before I take this medication?

They need to know if you have any of these conditions:

• Kidney disease
• An unusual or allergic reaction to miglustat, other medications, foods, dyes, or preservatives
• Pregnant or trying to get pregnant
• Breastfeeding

How should I use this medication?

Take this medication by mouth with an unsweetened beverage (water, tea or coffee with no cream, sugar, or sweeteners). Take it on an empty stomach, at least 2 hours before or 2 hours after other beverages or food. Take it as directed on the prescription label. Do not cut, crush, or chew this medication. Swallow the capsules whole. Keep taking it unless your care team tells you to stop.

Talk to your care team about the use of this medication in children. Special care may be needed.

Overdosage: If you think you have taken too much of this medicine contact a poison control center or emergency room at once.

NOTE: This medicine is only for you. Do not share this medicine with others.

What if I miss a dose?

It is important not to miss any doses. Talk to your care team about what to do if you miss a dose.

What may interact with this medication?

Interactions are not expected.

This list may not describe all possible interactions. Give your health care provider a list of all the medicines, herbs, non-prescription drugs, or dietary supplements you use. Also tell them if you smoke, drink alcohol, or use illegal drugs. Some items may interact with your medicine.

What should I watch for while using this medication?

Visit your care team for regular checks on your progress. Tell your care team if your symptoms do not start to get better or if they get worse.

Talk to your care team if you may be pregnant. Serious birth defects can occur if you take this medication during pregnancy. You will need a negative pregnancy test before starting this medication. Contraception is recommended while taking this medication and for 60 days after the last dose. Your care team can help you find the option that works for you.

Do not breastfeed while taking this medication.

This medication may cause infertility. Talk to your care team if you are concerned about your fertility.

What side effects may I notice from receiving this medication?

Side effects that you should report to your care team as soon as possible:

• Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

• Diarrhea
• Fatigue
• Fever
• Headache
• Nausea
• Stomach pain

This list may not describe all possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Where should I keep my medication?

Keep out of the reach of children and pets.

Store at room temperature between 20 and 25 degrees C (68 and 77 degrees F). Keep this medication in the original container. Protect from light. Get rid of any unused medication after the expiration date.

To get rid of medications that are no longer needed or have expired:

• Take the medication to a medication take-back program. Check with your pharmacy or law enforcement to find a location.
• If you cannot return the medication, check the label or package insert to see if the medication should be thrown out in the garbage or flushed down the toilet. If you are not sure, ask your care team. If it is safe to put it in the trash, empty the medication out of the container. Mix the medication with cat litter, dirt, coffee grounds, or other unwanted substance. Seal the mixture in a bag or container. Put it in the trash.

NOTE: This sheet is a summary. It may not cover all possible information. If you have questions about this medicine, talk to your doctor, pharmacist, or health care provider.