Miglustat

Miglustat is a prescription medication used as monotherapy for the treatment of mild-to-moderate type 1 Gaucher disease in adults for whom enzyme replacement therapy is not a therapeutic option.

• Miglustat is available under the following different brand names: Zavesca.

Common side effects of Miglustat include:

• flatulence
• constipation
• bloating
• nausea
• vomiting
• stomach pain
• indigestion
• loss of appetite
• weight loss
• dry mouth
• dizziness
• weakness
• headache (including migraine)
• back pain
• muscle cramps
• leg cramps
• pain or heavy feeling in the arms or legs
• memory problems
• feeling unsteady
• loss of balance
• burning pain in the hands or feet
• vision problems
• changes in the menstrual periods

Serious side effects of Miglustat include:

• hives
• difficulty breathing
• swelling of the face, lips, tongue, or throat
• tremor or uncontrolled shaking (especially in the hands)
• numbness or tingling in the hands or feet
• burning pain in the hands or feet
• severe diarrhea
• weight loss
• easy bruising
• unusual bleeding (nosebleeds, bleeding gums).

Rare side effects of Miglustat include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Capsule

• 100 mg

Gaucher Disease

Adult dosage

• 100 mg orally every 8 hours; decrease to once a day or every 12 hours for patients with adverse effects.

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Miglustat has severe interactions with no other drugs.
• Miglustat has serious interactions with no other drugs.
• Miglustat has moderate interactions with the following drug:
  • imiglucerase
• Miglustat has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• hypersensitivity to product or components.

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Miglustat?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Miglustat?”

Cautions

• Cases of peripheral neuropathy have been reported; patients should undergo baseline & repeat neuro evaluation at 6-month intervals; consider treatment cessation if symptoms develop
• Tremor may develop or be exacerbated; dose reduction may ameliorate, usually within days; discontinuation may be required
• Avoid high carbohydrate foods during treatment to lessen side effects of diarrhea; weight loss reported due to diarrhea; incidence decreases over time
• May affect spermatogenesis & sperm parameters; male patients should use reliable contraception
• May increase the clearance of Cerezyme (imiglucerase); concomitant use is not recommended
• Mild decrease in platelet counts reported; bleeding not reported
• Exacerbation of existing tremors may occur that may resolve over time or improve with dose reduction; may consider discontinuing therapy if it does not resolve

Pregnancy and Lactation

• Based on findings from animal reproduction studies, a drug may cause fetal harm when administered to a pregnant woman; available data from postmarketing case reports with drug use in pregnancy are insufficient to assess a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes; there are risks associated with symptomatic Type I Gaucher disease in pregnancy, including hepatosplenomegaly and thrombocytopenia; advise pregnant women of potential risks to the fetus
• Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations; Type 1 Gaucher disease manifestations may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of pregnancy, and thrombocytopenia which can lead to increased bleeding and possible hemorrhage.
• Reproductive potential
  • Findings from a small clinical study in seven healthy adult males who received therapy for six weeks did not indicate effects on male fertility; studies in male rats have shown that the drug decreased fertility, but findings were reversible; studies in female rats have shown increased post-implantation loss and decreased embryo-fetal survival
• Lactation
  • There are no available data on the presence of the drug in either human or animal milk, its effects on breastfed infants, or milk production; based on the physical properties of Miglustat, the drug is likely to be present in breast milk; because of potential for serious adverse reactions in breastfed infants, advise women that breastfeeding is not recommended