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Home > Drug Category > Gaucher Disease > Zavesca and Equivalent

Zavesca Information

Zavesca (Miglustat) is primarily used in treating Type 1 Gaucher disease. In this condition, the body lacks an enzyme required to degrade a specific lipid (glucosylceramide), resulting in its accumulation in the liver, spleen, and bones. Zavesca is typically used for adults with mild-to-moderate symptoms who cannot tolerate enzyme replacement therapy, the standard treatment, perhaps due to an allergy or difficulty accessing veins for IV infusions. It is, however, not recommended for individuals with severe symptoms of type 1 Gaucher disease. It may also be used to treat Niemann-Pick type C, a severe neurological disease characterized by the accumulation of lipids in the brain and other organs. Because Zavesca is a small-molecule drug, it can cross the blood-brain barrier, a significant advantage for treating neurological symptoms.

The active ingredient, miglustat, is a synthetic analogue of D-glucose and belongs to a class of drugs known as substrate-reduction therapy. It acts as a competitive, reversible inhibitor of the enzyme glucosylceramide synthase, which catalyzes the first step in glycosphingolipid synthesis. By inhibiting the production of these excess fats at the source, miglustat reduces the metabolic load to a level that the patient’s residual natural enzymes can realistically manage. This appears to stabilize organ size and improve blood counts, while its ability to cross the blood-brain barrier likely accounts for its unique role in slowing the neurological decline associated with Niemann-Pick Type C.

Miglustat has also been found effective in the treatment of Pompe disease, a genetic disorder in which the body can’t produce enough of the enzyme acid alpha-glucosidase. It is used exclusively in combination with the enzyme replacement therapy Pombiliti for adults with late-onset Pompe disease. In Pompe disease, miglustat acts as a pharmacological chaperone that binds to and stabilizes the infused replacement enzyme, thereby preventing its degradation during circulation. This structural support ensures that a higher concentration of functional enzyme reaches muscle cells, where it can release and degrade the toxic accumulation of glycogen.

Zavesca Side Effects

Like all medications, Zavesca can cause some side effects. Common side effects of this medication may include constipation, decreased appetite, diarrhea, dizziness, dry mouth, flu-like symptoms, gas, generalized weakness, headache, heartburn, muscle cramps, nausea, sensation of spinning, abdominal pain, unusual tiredness, vision problems, and vomiting. Contact your doctor if these mild side effects persist or worsen.

Stop taking this medicine and contact your doctor immediately if you experience any serious side effects, including the following:
• Allergic reactions such as breathing difficulties, body itching, body rash, skin blistering, swelling of the face, mouth, tongue, lips, or throat;
• New or worsening tremors;
• Numbness, burning, or tingling in the hands and feet;
• Seizures;
• Severe or worsening stomach or abdominal pain;
• Unusual bruising or bleeding;
• Weight loss;
• Worsening diarrhea

Inform your doctor about any other side effects you notice that are not listed here.

Zavesca Precautions

Do not take this medication if you are allergic to miglustat, other ingredients in Zavesca, or any other medicines. Before you start taking this medication, ensure that you inform your doctor about any prior or current medical conditions, including the following:
• Bleeding or blood disorders such as low platelets;
• Kidney disease;
• Liver disease

Avoid driving or operating machinery until you know how this medication affects you, as it can cause reduced alertness. This medication is not recommended for use in pregnant women as it can harm the fetus. Women of childbearing age must have a negative pregnancy test before starting and use effective contraception during treatment. Consult your doctor before using this medication while breastfeeding to determine its risks and benefits to you and your infant. Evidence from animal studies suggests that miglustat may affect sperm count and quality. Men are typically advised to use reliable contraception during treatment and for 3 months after their last dose.

Your physician will request regular blood tests before and during treatment with this medication to monitor its effects in your body. This medication is not recommended for use in children with type 1 Gaucher disease. Taking miglustat along with imiglucerase can lower the levels of both drugs in your system, making the treatment less effective. Therefore, before using this medication, inform your doctor about any other medications you are currently taking, including herbal medicines and supplements, to determine if it is safe to use along with miglustat.

This medication is available in capsules for oral use. Your doctor will determine the appropriate dose for you depending on your condition. You should take these doses between meals to help minimize potential stomach upset and diarrhea. Always swallow the capsules whole with water and never crush or chew them. Store this medication at room temperature, protect it from moisture, and keep it out of the reach of children.

Do I need a prescription to order Zavesca?

Yes, a prescription is required from your physician to order Zavesca.