Information about Zavesca (Miglustat)
Zavesca (Miglustat) is a medication primarily used to treat certain rare genetic disorders, particularly those associated with the buildup of specific substances in the body. It works by inhibiting the enzyme glucosylceramide synthase, which is responsible for the production of glucosylceramide, a fat-like substance that can accumulate in cells in certain disorders. Zavesca helps reduce the accumulation of these substances, offering treatment for conditions like Gaucher disease and Niemann-Pick type C disease.
Product Highlights
- Used to treat type 1 Gaucher disease in adult and pediatric patients who have mild to moderate forms of the condition and cannot use enzyme replacement therapy.
- Zavesca is also used to treat Niemann-Pick type C disease, a rare, inherited condition that affects the nervous system and can lead to organ enlargement and neurological complications.
Key Ingredient
Key Benefits
- By inhibiting glucosylceramide synthase, Zavesca helps reduce the accumulation of fat-like substances in cells, preventing damage to organs and tissues.
- For patients with Gaucher disease or Niemann-Pick type C, Zavesca can help improve symptoms, potentially enhancing the quality of life by reducing organ enlargement and neurological symptoms.
- Zavesca is an oral alternative for patients who cannot tolerate or do not have access to enzyme replacement therapy, especially in Gaucher disease.
- In Niemann-Pick type C, it can help reduce neurological symptoms such as ataxia and cognitive decline, though the effect may vary.
Direction of Use
- The typical dosage for adults and children (age-dependent) is 100 mg, taken orally, two to three times per day. The exact dosage should be based on the patient's condition and response to the medication, as determined by their healthcare provider.
- Take Zavesca capsules with food to minimize stomach upset. Take the capsules whole with a full glass of water. Do not chew or break the capsules.
- Regular follow-up visits with a healthcare provider are necessary to monitor the drug's effectiveness and potential side effects.
Safety Concerns
- May include diarrhea, weight loss, nausea, abdominal pain, and flatulence. Some patients may experience fatigue or tremors.
- Rare but potentially serious side effects include liver problems, severe diarrhea, and signs of neurological impairment. Patients should be monitored for changes in liver function and gastrointestinal symptoms.
- Zavesca is classified as a pregnancy category C drug, meaning it may harm a fetus. It should only be used during pregnancy if the potential benefits exceed the risks. Miglustat is not recommended during breastfeeding due to potential risks to the infant.
- Zavesca should be used with caution in patients with liver problems, as it may require dose adjustments or close monitoring.
- Renal function should be monitored, as Zavesca may cause issues in patients with kidney conditions.
Avoid Zavesca (Miglustat) If
- Do not use Zavesca if you have a known hypersensitivity to miglustat or any of its components.
- Avoid Zavesca if you have severe liver disease, as it may increase the risk of liver toxicity.
- If you are pregnant or breastfeeding, consult with your healthcare provider before using Zavesca to assess the risks and benefits.
- In patients with severe kidney impairment, Zavesca may not be suitable or may require careful dosing and monitoring.
- Zavesca may worsen gastrointestinal issues such as diarrhea or malabsorption; it should be avoided in patients with severe GI disorders.
