What is miglustat?
Miglustat may reduce the formation of the protein glucosylceramide in the body. Individuals with type 1 Gaucher disease (a hereditary disorder) are missing an enzyme to break down glucosylceramide in the body. Therefore, it may build up in the tissues and cause problems. Untreated Gaucher disease may result in anemia (low level of red blood cells), thrombocytopenia (low level of platelets), bone disease, hepatomegaly (enlargement of the liver), or splenomegaly (enlargement of the spleen).
Miglustat is used in the treatment of mild to moderate type 1 Gaucher disease by people who cannot receive enzyme replacement therapy.
Miglustat may also be used for purposes other than those listed here.
Before taking miglustat, tell your doctor if you have kidney problems, any other medical conditions, or if you take any other medicines. You may not be able to take miglustat, or you may require a dosage adjustment or special monitoring during treatment.
Miglustat is in the FDA pregnancy category X. This means that miglustat is known to
cause birth defects in an unborn baby.
It is not known whether miglustat passes into breast milk and if it will affect a nursing baby. Do not take miglustat without first talking to your doctor if you are breast-feeding a baby.
Take miglustat exactly as directed by your doctor. If you do not understand these directions, ask your pharmacist, nurse, or doctor to explain them to you.
Take miglustat at the same time or times each day as directed by your doctor.
Swallow each capsule whole with a full glass of water.
Miglustat can be taken with or without food.
It is important to take miglustat regularly to get the most benefit.
Your doctor may want you to have neurological tests or other medical evaluations during treatment with miglustat to monitor progress and side effects.
Store miglustat at room temperature away from moisture and heat.
If you miss a dose of miglustat, skip the missed dose and only take the next regularly scheduled dose. Do not take a double dose of this medication unless otherwise directed by your doctor.
Nerve problems such as numbness, tingling, and tremor (shaking) of the hands have been reported by some people taking miglustat. Contact your doctor immediately if you experience new or worsening numbness, tingling, or burning in the hands, arms, legs, or feet or tremor in the hands.
Seek emergency medical attention if you experience a rare but serious allergic reaction to miglustat including difficulty breathing; closing of the throat; swelling of the lips, tongue, or face; or hives.
Other less serious side effects may be more likely to occur. Continue to take miglustat and talk to your doctor if you experience:
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diarrhea;
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decreased appetite or weight loss;
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gas or abdominal pain;
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constipation;
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muscle cramps or weakness;
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headache;
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dizziness; or
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vision problems.
Side effects other than those listed here may also occur. Talk to your doctor about any side effect that seems unusual or that is especially bothersome.
Nerve problems such as numbness, tingling, and tremor (shaking) of the hands have been reported by some people taking miglustat. Contact your doctor immediately if you experience new or worsening numbness, tingling, or burning in the hands, arms, legs, or feet or tremor in the hands.
There are no restrictions on food, beverages, or activity during treatment with miglustat unless otherwise directed by your doctor.
Miglustat is not intended for use with imiglucerase (Cerezyme), another treatment for type 1 Gaucher disease.
There are no known interactions between miglustat and other medications. Talk to your doctor or pharmacist before taking any other prescription or over-the-counter medications, including vitamins, minerals, and herbal products, during treatment with miglustat.
Seek emergency medical attention if an overdose is suspected.
Symptoms of a miglustat overdose are not well known but may include dizziness, numbness and tingling, and blood problems.