Miglustat (Oral)

Miglustat is given to people who have mild to moderate type 1 Gaucher disease.

Overview

People who suffer from Gaucher disease do not produce an enzyme that breaks down a chemical called glucosylceramide. This causes levels of glucosylceramide to increase in the body, which can cause damage to the liver, spleen, and bones.

The usual front-line treatment for type 1 Gaucher disease is enzyme replacement therapy, to give the patients the enzyme that they lack. However, this is not suitable for some individuals, perhaps due to allergies, hypersensitivity, or poor access to veins. In such people, miglustat is prescribed instead. Miglustat works by blocking the action of another enzyme called 'œglucosylceramide synthase'. This is the enzyme that creates glucosylceramide, so by stopping this enzyme from working miglustat reduces the amount of glucosylceramide in the body.

Miglustat can cause side effects in some people, the most common of which being diarrhea and tremors. If you already experience diarrhea or tremors, miglustat may make these symptoms worse.

Miglustat is sold under the brand name 'œZavesca.'

Conditions Treated

  • Type 1 Gaucher disease

Type Of Medicine

  • Enzyme inhibitor

Side Effects

Miglustat is known to cause side effects in some people. These are listed below.

Diarrhea and weight loss

Diarrhea, usually accompanied by weight loss, is the most common side effect of miglustat and affects 65-85% of the people who take it. The diarrhea tends to reduce over time, and your doctor may recommend changes to your diet, such as a reduction in sucrose, lactose, and other carbohydrates, or they may prescribe you an anti-diarrheal medication to take with miglustat. Consult your doctor right away if you experience diarrhea or weight loss while taking miglustat.

Tremors

Around 30% of people taking miglustat reported tremors or shaking in the hands. This most commonly occurs in the first month of treatment with miglustat and usually disappears by itself within 1 to 3 months as your body adjusts to the medication. Contact your doctor immediately if you experience tremors. They may ask you to reduce your dose to see if this removes the tremors, or they may ask you to stop taking the medication.

Peripheral neuropathy

This is a condition in which the nerves of the body have been damaged, which prevents them sending signals as well as they normally would. It most commonly affects the hands and the feet, but it may affect other parts of the body also. Symptoms of peripheral neuropathy are seen in around 3% of people taking miglustat. Consult your doctor immediately if you notice any symptoms of peripheral neuropathy, which include:

  • Burning sensations

Other side effects you should tell your doctor about

In addition to the above, you should contact your doctor immediately if you experience any of the symptoms listed below:

  • Any unusual bleeding, but especially in the gums or mouth

Less serious side effects

Miglustat can also cause some other side effects that are less serious and do not usually require medical attention. In some cases, they will go away on their own after some time. Contact your doctor if you experience any of the side effects below, especially if they persist over time, or if you are concerned about them.

This list is not exhaustive, and you might experience different side effects while you are taking miglustat. If you notice any other side effects that are unusual or that concern you, contact your doctor for advice.

Overdose

The symptoms of overdosing on miglustat are not well-known; however, they may include clumsiness, unsteadiness, feeling unbalanced, or a lack of coordination. Seek medical attention immediately if you believe you have overdosed on miglustat.

Allergic reactions

Serious allergic reactions to miglustat are rare, but they can potentially occur. You may also be allergic to the non-active ingredients in the capsules. Tell your doctor about anything you are allergic to before you are prescribed miglustat. Seek emergency medical attention if you experience the symptoms of an allergic reaction, which include:

  • Extreme dizziness, disorientation, drowsiness, or tiredness

Dosage

Your doctor will prescribe a dose of miglustat based on your symptoms, age, the medical conditions you have, and other factors. You should take the medication exactly as prescribed by your doctor. Do not take a higher dose than prescribed, and do not take the medication for longer than you have been advised. With that said, here are some common dosages:

  • Adults: 100 mg three times every day at regular intervals

You will normally be advised to take miglustat on an empty stomach or between meals, as this will reduce the risk of diarrhea.

If you miss one of your doses, such as if you forget or are unable to take it, skip the dose and continue from your next scheduled dose as normal. Do you take a double dose to compensate for a missed one, as this may place you at higher risk of experiencing side effects.

Interactions

Some medications may interact with miglustat in ways that produce undesired side effects. Before you take miglustat, make your doctor aware of any other medications, vitamins, or herbal supplements that you are taking. The following medication is of particular concern:

  • Imiglucerase (known by the brand name Cerezyme)

Imiglucerase is another drug that is prescribed to treat Gaucher disease. Miglustat may interfere with this medication in a way that reduces its effectiveness. If you are currently taking imiglucerase, this does not mean that you cannot take miglustat. However, if you do, your doctor may want to adjust your dose of one or both medications, and you will probably be asked to attend check-up appointments more frequently. If you notice any change in your symptoms while taking miglustat, consult your doctor without delay.

Warnings

Miglustat may cause additional problems if it is taken by people who have certain existing medical conditions. Make sure you discuss any other medical conditions you suffer from with your doctor, so they can determine whether miglustat is safe for you to take. In particular, tell your doctor if you have any of the conditions listed below, or if you have a history of them:

  • Diarrhea: this medication can cause diarrhea as a side effect, so people who already have this should use this drug with caution.

With the exception of severe kidney disease, the presence of these conditions does not necessarily mean you can't take miglustat. However, your doctor may want to give you a different dose, and they may want to see you more often for checkups.

In rare cases, miglustat can cause problems with the blood (lower levels of platelets in the blood), and the nerves (peripheral neuropathy). Your doctor will probably want to make appointments for you to undergo blood tests and neurological exams regularly. Make sure you keep these appointments as this will help your doctor to determine whether any adverse effects are occurring.

Pregnancy

Animal studies have shown that miglustat can be harmful to the unborn fetus, but it is not known whether this is also true in humans. Pregnant women are usually not advised to take miglustat unless the potential benefit of taking it would outweigh the potential risks. If you become pregnant while you are taking miglustat, contact your doctor immediately.

Breastfeeding

This medication enters the breastmilk and can, therefore, pass to the infant during breastfeeding. You should not breastfeed while taking this medication.

Elderly people

Studies have shown that miglustat is effective as a treatment in older individuals; however, this demographic is more likely to have existing liver or kidney problems. If you are over the age of 65, your doctor will probably ask to see you more often for check-ups.

Storage

Improper storage of your miglustat capsules can make them less potent or even dangerous to take. Observe the following instructions when storing your medication.

Store at room temperature:

Keep the capsules well away from sources of heat. Do not refrigerate the tablets, and do not store them in the freezer. Do not keep them in the bathroom as the shower makes this room too warm to safely store medications.

Store in a dry location:

Keep the capsules away from sources of humidity or moisture. Do not store this medication in the bathroom.

Avoid light:

Do not store this medicine near a window or in any other location where it will be exposed to direct sunlight.

Keep away from children:

Miglustat may be harmful when consumed by children. Store the medication in a child-proof container, and out of the sight and reach of children.

Summary

Miglustat capsules are given to people with Gaucher disease who are not able to receive enzyme replacement therapy.

Miglustat is known to produce unpleasant side effects in many people who take it. The most common side effect is diarrhea, which affects over two-thirds of patients, followed by hand tremors, affecting around a third of patients. Taking the medication on an empty stomach can help reduce the risk of diarrhea.