Miglustat (mi-GLOO-stat)

Treats type I Gaucher disease and Pompe disease.

Brand Name(s):

Opfolda, Yargesa, Zavesca

When This Medicine Should Not Be Used:

How to Use This Medicine:

Capsule

• Take your medicine as directed. Your dose may need to be changed several times to find what works best for you.
• Opfolda™:
  • Swallow the capsule whole with unsweetened drinks (including water, tea, or coffee with no cream, sugar, or sweeteners). Do not eat or drink for at least 2 hours before and 2 hours after taking this medicine.
  • Take this medicine 1 hour before receiving Pombiliti™ injection.
• Read and follow the patient instructions that come with this medicine. Talk to your doctor or pharmacist if you have any questions.
• Missed dose:
  • Opfolda™: If you miss a dose, you should not receive Pombiliti™ injection. If you miss this medicine in combination with Pombiliti™ injection, restart treatment as soon as possible.
  • Zavesca®: If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
• Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

• Some medicines can affect how miglustat works. Tell your doctor if you are using imiglucerase.

Warnings While Using This Medicine:

• It is not safe to take this medicine during pregnancy. It could harm an unborn baby. Tell your doctor right away if you become pregnant. If you are woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before receiving this medicine. Use an effective form of birth control during treatment and for at least 60 days after the last dose.
• Do not breastfeed during treatment with this medicine.
• Tell your doctor if you have kidney disease, heart disease, lung or breathing problems, tremors, or a low number of platelets in your blood.
• This medicine may cause the following problems:
  • Peripheral neuropathy (a nerve problem)
  • Tremors
  • Diarrhea
• This medicine could cause infertility. Talk with your doctor before using this medicine if you plan to have children.
• Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments.
• Keep all medicine out of the reach of children. Never share your medicine with anyone.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

• Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
• Numbness, tingling, or burning pain in your hands, arms, legs, or feet
• Severe diarrhea, nausea, or vomiting
• Trembling or shaking of the hands (either new or worse than usual)
• Unusual bleeding, bruising, or weakness

If you notice these less serious side effects, talk with your doctor:

• Fever, headache, or dizziness
• Mild nausea, vomiting, constipation, stomach pain or upset
• Muscle cramps
• Tiredness
• Weight loss or loss of appetite

If you notice other side effects that you think are caused by this medicine, tell your doctor

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088