Zavesca (miglustat) Gaucher Disease Drug Side Effects & Warnings


Generic drug: miglustat

Brand name: Zavesca

What is Zavesca (miglustat), and how does it work?

Zavesca (miglustat) is a prescription medicine used alone to treat adults with mild to moderate type 1 Gaucher disease. Zavesca is used only in people who cannot be treated with enzyme replacement therapy.

It is not known if Zavesca is safe and effective in children under 18 years of age.

What are the side effects of Zavesca?

Zavesca may cause serious side effects including:

  • Numbness, tingling, pain, or burning in your hands or feet (peripheral neuropathy). Call your healthcare provider right away if you get numbness, tingling, pain, or burning in your hands or feet.
  • Your healthcare provider may test your nerves (neurological exam) before you start Zavesca and during treatment with Zavesca.
  • New or worsening hand tremors (shaky movements). Tremors are common with Zavesca and may begin within the first month of starting treatment. Sometimes the tremors may go away between 1 to 3 months with continued treatment. Your healthcare provider may lower your dose or stop Zavesca if you develop new or worsening hand tremors. Call your healthcare provider right away if you get new hand tremors during treatment with Zavesca or if the hand tremors you already have get worse.
  • Diarrhea is common with Zavesca and sometimes can be serious. Your healthcare provider may prescribe another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you and may recommend changes to your diet, such as avoiding foods high in carbohydrates. Talk with your healthcare provider about your diet if you have diarrhea.
  • Weight loss is common with Zavesca and sometimes can be serious. You may lose weight when you start treatment with Zavesca.
  • Low platelet count is common with Zavesca and can be serious. Your healthcare provider may do blood tests to monitor your blood platelet count during treatment with Zavesca.

The most common side effects of Zavesca include:

These are not all of the possible side effects of Zavesca.

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

What is the dosage for Zavesca?

Instructions For Administration

  • Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease.
  • The recommended dose for the treatment of adult patients with type 1 Gaucher disease is one 100 mg capsule administered orally three times a day at regular intervals. If a dose is missed, the next
    Zavesca capsule should be taken at the next scheduled time.
  • It may be necessary to reduce the dose to one 100 mg capsule once or twice a day in some patients due to adverse reactions, such as tremor or diarrhea.

Patients With Renal Insufficiency

  • In patients with mild renal impairment (adjusted creatinine clearance 50-70 mL/min/1.73 m2), initiate
    Zavesca treatment at a dose of 100 mg twice per day.
  • In patients with moderate renal impairment (adjusted creatinine clearance of 30-50 mL/min/1.73 m2), initiate
    Zavesca treatment at a dose of one 100 mg capsule per day.
  • Zavesca is not recommended for use in patients with severe renal impairment (creatinine clearance <30 mL/min/1.73 m2).

What drugs interact with Zavesca?

While co-administration of Zavesca appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase.

Is Zavesca safe to use while pregnant or breastfeeding?

  • Based on findings from animal reproduction studies, Zavesca may cause fetal harm when administered to a pregnant woman.
  • Available data from postmarketing case reports with Zavesca use in pregnancy are insufficient to assess a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
  • There are risks associated with symptomatic Type I Gaucher disease in pregnancy, including hepatosplenomegaly and thrombocytopenia.
  • There are no available data on the presence of miglustat in either human or animal milk, the effects on the breastfed infant, or the effects on milk production.
  • Based on the physical properties of miglustat, Zavesca is likely to be present in breast milk.
  • Because of the potential for serious adverse reactions in breastfed infants, advise women that breastfeeding is not recommended.

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