Patient information: uses, side effects, how to take - Miglustat
What are Miglustat Capsules?
Miglustat capsules are a prescription medicine used alone to treat adults with mild to moderate type 1 Gaucher disease. Miglustat capsules are used only in people who cannot be treated with enzyme replacement therapy.
It is not known if miglustat capsules are safe and effective in children under 18 years of age.
Before taking Miglustat Capsules, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems
are pregnant or plan to become pregnant. Miglustat capsules may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with miglustat capsules.
- are breastfeeding or plan to breastfeed. It is not known if miglustat can pass into your breast milk and may harm your baby. Do not breastfeed during treatment with miglustat capsules. Talk to your healthcare provider about the best way to feed your baby during treatment with miglustat capsules.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Miglustat capsules may affect how other medicines work.
How should I take Miglustat Capsules?
- Take miglustat capsules exactly as your healthcare provider tells you to.
- Take miglustat capsules at the same time each day.
- If you miss a dose of miglustat capsules, skip that dose. Take the next miglustat capsule at the usual time.
What are the possible side effects of Miglustat Capsules?
Miglustat Capsules may cause serious side effects including:
- Numbness, tingling, pain, or burning in your hands or feet (peripheral neuropathy). Call your healthcare provider right away if you get numbness, tingling, pain, or burning in your hands or feet.
- Your healthcare provider may test your nerves (neurological exam) before you start miglustat and during treatment with miglustat capsules.
- New or worsening hand tremors (shaky movements). Tremors are common with miglustat and may begin within the first month of starting treatment. Sometimes the tremors may go away between 1 to 3 months with continued treatment. Your healthcare provider may lower your dose or stop miglustat capsules if you develop new or worsening hand tremors. Call your healthcare provider right away if you get new hand tremors during treatment with miglustat capsules or if the hand tremors you already have get worse.
- Diarrhea is common with miglustat and sometimes can be serious. Your healthcare provider may prescribe another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you and may recommend changes to your diet, such as avoiding foods high in carbohydrates. Talk with your healthcare provider about your diet if you have diarrhea.
- Weight loss is common with miglustat and sometimes can be serious. You may lose weight when you start treatment with miglustat capsules.
- Low platelet count is common with miglustat and can be serious. Your healthcare provider may do blood tests to monitor your blood platelet count during treatment with miglustat capsules.
The most common side effects of miglustat capsules include:
- Weight loss
- Stomach pain
- Gas
- Nausea and vomiting
- Headache, including migraine
- Back pain
- Constipation
- Dry mouth
- Heaviness in arms and legs
- Memory loss
- Unsteady walking
- Leg cramps
- Dizziness
- Weakness
- Vision problems
- Muscle cramps
- Loss of appetite
- Indigestion
- Numbness, tingling, pain, or burning of your skin
- Stomach bloating
- Stomach pain not related to food
- Menstrual changes
These are not all the possible side effects of miglustat capsules.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
How should I store Miglustat Capsules?
- Store miglustat capsules at room temperature between 68ºF to 77ºF (20ºC to 25ºC).
Keep miglustat capsules and all medicines out of the reach of children.
General information about the safe and effective use of miglustat capsules.
Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use miglustat capsules for a condition for which it was not prescribed. Do not give miglustat capsules to other people, even if they have the same symptoms you have. It may harm them.
You can ask your healthcare provider or pharmacist for information about miglustat capsules that is written for health professionals.
What are the ingredients in Miglustat Capsules?
Active ingredient: miglustat
Inactive ingredients: magnesium stearate.
The capsule shell contains: gelatin, sodium lauryl sulfate and titanium dioxide; the edible printing ink contains black iron oxide, propylene glycol, potassium hydroxide and shellac.
Manufactured by: ANI Pharmaceuticals, Inc. Baudette, MN 56623
For more information about miglustat capsules call 1-855-204-1431 or go to www.anipharmaceuticals.com.
This Patient Information has been approved by the U.S. Food and Drug Administration.
Revised: 06/2025