Miglustat

WHY is this medicine prescribed?

Miglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems). Miglustat is also used in combination with another medication known as cipaglucosidase alfa-atga (Pombilti) to treat late onset Pompe disease (a genetic condition that causes deficiency in lysosomal acid alpha-glucosidase, which can lead to muscle weakness and wasting) in adult patients who weigh 92 pounds (40 kg) or more and who have not been improving on their enzyme therapy. Miglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from producing the fatty substance so that less of it will build up in the body and cause symptoms.

HOW should this medicine be used?

Miglustat comes as a capsule to take by mouth. For treatment of Gaucher disease (Zavesca), it is usually taken with or without food and with plenty of water up to three times a day. For treatment of Pompe disease (Opfolda), it is usually taken every other week approximately 1 hour before the planned administration of cipaglucosidase alfa-atga (Pombilti). Take it at around the same time(s) every day. For Pompe disease, miglustat (Opfolda) should be taken with unsweetened beverages such as water, coffee, or tea (with no sugar, cream or sweeteners) at least 2 hours before or after eating or drinking any other beverage. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take miglustat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole; do not split, chew, or crush them.

Miglustat controls Gaucher and Pompe disease but does not cure it. Continue to take miglustat even if you feel well. Do not stop taking miglustat without talking to your doctor.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What SPECIAL PRECAUTIONS should I follow?

Before taking miglustat,

• tell your doctor and pharmacist if you are allergic to miglustat, any other medications, or any of the ingredients in miglustat. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take.
• tell your doctor if you have or have ever had a tremor (shaking of your hands that you cannot control); pain, burning, tingling, or numbness in your hands or feet; any disease that affects your nervous system; or kidney disease.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. You should use effective birth control to prevent pregnancy while you are taking miglustat and for 60 days after your last dose. If you become pregnant while taking miglustat, call your doctor immediately. Miglustat may harm the fetus. Breastfeeding while receiving miglustat is not recommended.
• you should know that miglustat may damage sperm. Men should continue to use effective birth control.

What SPECIAL DIETARY instructions should I follow?

Miglustat may cause diarrhea and weight loss. If you experience these side effects, your doctor will tell you how to change your diet to improve your symptoms. You may be told to avoid foods that are high in carbohydrates. Follow these instructions carefully.

What should I do IF I FORGET to take a dose?

If taking miglustat for Gaucher disease (Zavesca), skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one. If taking miglustat for Pompe disease (Opfolda) and you miss the dose of miglustat, you should call and reschedule your cipaglucosidase alfa-atga and then take the miglustat dose an hour before the newly scheduled cipaglucosidase alfa-atga.

What SIDE EFFECTS can this medicine cause?

Miglustat may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• diarrhea
• stomach pain or bloating
• gas
• loss of appetite
• weight loss
• upset stomach
• vomiting
• constipation
• indigestion
• dry mouth
• weakness
• muscle cramps, especially in the legs
• feeling of heaviness in the arms or legs
• unsteadiness when walking
• back pain
• dizziness
• nervousness
• headache
• memory problems
• difficult or irregular menstruation (period)
• tiredness
• fever

Some side effects can be serious. The following symptoms are uncommon, but if you experience any of them, call your doctor immediately:

• pain, burning, numbness or tingling in your hands, arms, legs, or feet
• shaking hands that you cannot control
• changes in vision
• easy bruising or bleeding

Miglustat may cause other side effects. Call your doctor if you have any unusual problems while taking this medication

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I know about STORAGE and DISPOSAL of this medication?

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (https://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location — one that is up and away and out of their sight and reach. https://www.upandaway.org

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor. Keep all appointments for cipaglucosidase alfa-atga infusion if you are receiving miglustat for Pompe disease.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.

The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.

AHFS^® Patient Medication Information™. © Copyright, 2024. The American Society of Health-System Pharmacists^®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.

Selected Revisions: December 15, 2023.