miglustat
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.
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.
Drug facts
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%)
Less Common 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
The following Miglustat (Zavesca) 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
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
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
excess air or gas in the stomach or intestines
halos around lights
headache
heaviness in the limbs
indigestion
leg cramps
loss of appetite
memory loss
menstrual changes
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.
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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.
Common questions about Miglustat (Zavesca)
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.
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.
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.
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.
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.
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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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
Typical dosage 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
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.
How much does Miglustat (Zavesca) cost?
$0.00
What are alternatives to Miglustat (Zavesca)?
Miglustat (Zavesca) images
