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miglustat

Generic Zavesca

Used for Lysosomal Storage Disease

Miglustat (Zavesca) is an oral medication used to treat adults with mild to moderate type 1 Gaucher disease. It’s only used in people who can’t receive enzyme replacement therapy (ERT). Miglustat (Zavesca) is available as a generic medication so it might be less expensive than other branded medications approved for this condition.

Reviewed by:

Jenny Zhang, PharmD, BCOP

Daisy Chau, PharmD

Last reviewed on March 15, 2022

Basics Side effects Pros & cons Pharmacist tips FAQs Warnings Dosage Interactions Alternatives Prices Medicare

What is Miglustat (Zavesca)?

What is Miglustat (Zavesca) used for?

Mild to moderate type 1 Gaucher disease in adults

How Miglustat (Zavesca) works

Miglustat (Zavesca) is a glucosylceramide synthase inhibitor. People with Gaucher disease don't have enough of an enzyme, a specific protein, that breaks down fatty substances (glycosphingolipids) called glucosylceramides. As a result, there are higher levels of glucosylceramide in the body, and it can build up in the spleen, liver, and bones. Miglustat (Zavesca) helps to lower the amount of glucosylceramide in your body by blocking it from being made.

Are you looking for information on Cerdelga (eliglustat) instead?

Drug Facts

Common BrandsZavesca, Yargesa

Drug ClassGlucosylceramide synthase inhibitor

Controlled Substance ClassificationNot a controlled medication

Generic StatusLower-cost generic available

AvailabilityPrescription only

What are the side effects of Miglustat (Zavesca)?

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Diarrhea (89-100%)
Weight loss (39-67%)
Stomach pain (18-67%)
Passing gas (29-50%)
Tremor (11-30%)
Headache (21-22%)
Nausea (8-22%)
Weakness (17%)
Vision problems (<17%)
Vomiting (4-11%)
Leg cramps (4-11%)

Other Side Effects

Bloating
Loss of appetite
Heartburn
Dizziness
Migraine
Muscle cramps
Menstrual problems
Constipation
Dry mouth
Back pain
Heaviness in limbs
Unstable when walking
Memory loss

Source: DailyMed

The following side effects have also been reported

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Black, tarry stools

bleeding gums

blood in the urine or stools

burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings

diarrhea

pinpoint red spots on the skin

trembling or shaking in the hands

unsteadiness or awkwardness

unusual bleeding or bruising

weakness in the arms, hands, legs, or feet

weight loss

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Back pain

bloated or full feeling

blurred or loss of vision

difficulty having a bowel movement (stool)

disturbed color perception

dizziness

double vision

dry mouth

excess air or gas in the stomach or intestines

halos around lights

headache

heartburn

heaviness in the limbs

indigestion

leg cramps

loss of appetite

memory loss

menstrual changes

nausea

pain or discomfort in the chest, upper stomach, or throat

passing gas

stomach discomfort, upset or pain

swelling of the abdominal or stomach area

tunnel vision

unsteady walk

vomiting

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Pros and cons of Miglustat (Zavesca)

Pros

Available as a lower-cost generic medication

More convenient than frequent trips to the infusion center for ERT

Is available as an oral medication, so it doesn’t require an IV or port compared to ERT

Doesn't require genetic testing before starting like Cerdelga (eliglustat)

Cons

Diarrhea and tremors are very common side effects

Is taken three times a day in most adults

Not for use in children or teenagers with type 1 Gaucher disease

Can only be used if you can’t receive ERT

Pharmacist tips for Miglustat (Zavesca)

Take miglustat (Zavesca) by mouth three times a day, or about every 8 hours. It can be taken with or without food. Spacing miglustat (Zavesca) from meals can help with diarrhea, but the opposite effect has also been seen in some people.

Try to take miglustat (Zavesca) around the same times every day. If you miss a dose, skip it and take your next scheduled dose at your usual time.

Miglustat (Zavesca) can cause new or worsening tremors, usually in the hands. Let your provider know if you have bothersome tremors or tremors that don't improve within a few months after starting this medication. In some cases, your provider might need to lower your dose, or you might have to stop taking miglustat (Zavesca) altogether.

Diarrhea is a very common side effect of miglustat (Zavesca). Luckily, it can be improved by changes in your diet. It's recommended to eat fewer foods that contain sugar, dairy, and carbohydrates while taking miglustat (Zavesca). Diarrhea should also improve over time. Let your provider know if you have worsening or bothersome diarrhea.

Frequently asked questions about Miglustat (Zavesca)

What is Gaucher disease?

Gaucher disease is a rare, genetic condition where your body has lower levels of an enzyme, a specific protein, that breaks down a type of fatty substance called glucosylceramide (a type of glycosphingolipid). As a result, glucosylceramide can build up in certain organs, like your spleen, liver, and bones. A buildup in the liver or spleen can lead to these organs getting abnormally large and causing a swollen belly. While a buildup in the bones and bone marrow can cause serious bleeding, bruising, and bone pain or weakness. Miglustat (Zavesca) helps to lower the amount of glucosylceramide in your body by blocking it from being made.

How is miglustat (Zavesca) different from enzyme replacement therapy (ERT)?

As mentioned above, in Gaucher disease, your body doesn't have enough of an enzyme that breaks down glucosylceramide. This leads to glucosylceramide collecting in different parts of the body, which can cause serious complications. ERT (e.g. Cerezyme (imiglucerase), VPRIV (velaglucerase alfa), or Elelyso (taliglucerase alfa)) works by replacing the enzyme that your body is missing. Then, your body can break down glucosylceramide appropriately. ERT is given by an infusion into your veins (intravenously, IV) roughly every 2 weeks. Miglustat (Zavesca) is an oral medication for Gaucher disease. It works by blocking glucosylceramide from being made. This then lowers the amount of glucosylceramide in your body so that it can't build up. It can be used alone when ERT is not a good option, due to allergy, intolerance, or difficulty with IV access. Talk to your provider about these options to find the best one for you.

Can you use miglustat (Zavesca) if you've already been treated with ERT before?

Yes, miglustat (Zavesca) was studied in people who have never received ERT and also in people who have previously been treated with Cerezyme (imiglucerase), a kind of ERT. In the studies where people had been treated with ERT before, ERT was stopped for at least 6 months before starting miglustat (Zavesca). Talk to your provider if you are interested in switching to ERT.

What's the difference between miglustat (Zavesca) and Cerdelga (eliglustat)?

Miglustat (Zavesca) and Cerdelga (eliglustat) are the only oral medications currently FDA approved to treat type 1 Gaucher disease. They both work the same way by blocking the production of glucosylceramide, which lowers the amount of glucosylceramide in your body. While Cerdelga (eliglustat) can be used as an alternative to ERT right away, miglustat (Zavesca) can only be used in people who can’t receive ERT for a specific reason (e.g. allergy, intolerance, or difficulty with IV access). Cerdelga (eliglustat) is taken either once or twice a day, while miglustat (Zavesca) is taken three times a day in most adults. Cerdelga (eliglustat) has many more interactions with other medications and supplements compared to miglustat (Zavesca). They also have different side effects so it's important to talk with your provider to find the best option for you.

Can you take miglustat (Zavesca) if you have kidney problems?

Make sure to let your provider know if you have kidney problems since you might need a different dose of miglustat (Zavesca). The medication is removed from your body through your kidneys. In people with kidney problems, the amount of this medication in your body can be higher and cause more side effects. Miglustat (Zavesca) is not recommended if you have severe kidney problems.

Is miglustat (Zavesca) safe in pregnancy or breastfeeding?

Miglustat (Zavesca) is not recommended for people who are pregnant or breastfeeding because there isn't enough information in humans. In fact, females with type 1 Gaucher disease have a higher risk of miscarriage, especially if symptoms aren't controlled before and during pregnancy. Pregnancy can also worsen those symptoms. Animal studies suggest that miglustat (Zavesca) can lead to birth defects. Also, it can likely be passed along in human milk, which can cause serious side effects in breastfed babies. If you're pregnant, think you might become pregnant, or breastfeeding, talk to your provider before taking miglustat (Zavesca).

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What are the risks and warnings for Miglustat (Zavesca)?

Miglustat (Zavesca) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

Nerve problems (peripheral neuropathy)

Miglustat (Zavesca) can cause nerve problems, or peripheral neuropathy. This can affect your arms, hands, legs, and feet. Your provider will perform a neurologic exam before you start this medication, and about twice a year during treatment. Let your provider know if you experience numbness, burning, a feeling of "pins and needles", tremors, problems with balance, or weakness in your arms or legs. You might need to stop taking miglustat (Zavesca) depending on your symptoms.

Tremors

Risk factors: History of tremor

Miglustat (Zavesca) can commonly cause new or worsening tremors, typically in the hands. This usually begins within the first month of treatment and goes away after a few more months of taking this medication. Depending on your symptoms, your provider might need to lower your dose or have you stop taking miglustat (Zavesca) altogether.

Diarrhea and weight loss

Miglustat (Zavesca) commonly causes diarrhea and weight loss. Miglustat (Zavesca) affects the way your intestines absorb sugars from your diet. When not enough sugars are absorbed, more water is drawn into the intestines and causes diarrhea. The diarrhea improves over time while taking this medication. Changing your diet (eating fewer foods that contain sugar, dairy, or carbohydrates), taking miglustat (Zavesca) in between meals, or taking anti-diarrheal medications like loperamide (Imodium A-D) can also help prevent diarrhea. Let your provider know if you continue to have diarrhea that doesn't improve with time or changes in your diet.

You might start losing weight while taking miglustat (Zavesca), especially during the first year of treatment. It's unclear whether this weight loss is due to the diarrhea or other stomach issues, eating less food, or a combination of these factors.

Lowering of platelet count

Risk factors: History of low platelet counts

Miglustat (Zavesca) can lower your platelet counts. This is generally mild and doesn’t usually result in bleeding. Your provider will regularly check your blood counts to monitor your platelets.

Miglustat (Zavesca) dosage forms

Typical dosing for Miglustat (Zavesca)

The typical dose is 100 mg by mouth three times daily, or about every 8 hours. Your dose might differ if you have kidney problems.

Interactions between Miglustat (Zavesca) and other drugs

Miglustat (Zavesca) may interact with certain medications or supplements. Always let your doctor and pharmacist know about any other medications or supplements (including prescribed and over-the-counter medications, vitamins, and dietary or herbal supplements) that you are currently taking. The list below does not include all possible drug interactions with Miglustat (Zavesca). Please note that only the generic name of each medication is listed below.

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.