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Actelion Pharmaceuticals US, Inc.
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South San Francisco, CA 94080
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ZAVESCA (miglustat) is indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (eg, due to constraints such as allergy, hypersensitivity, or poor venous access).
DOSAGE AND ADMINISTRATION
Instructions for Administration
- The recommended dose for the treatment of adult patients with type 1 Gaucher disease is 100 mg capsule administered orally three times a day at regular intervals. It may be necessary to reduce the dose to one 100 mg capsule once or twice a day in some patients for adverse effects, such as diarrhea or tremor.
Patients with Renal Insufficiency
- In patients with mild renal impairment (adjusted creatinine clearance 50-70 mL/min/1.73 m2), ZAVESCA administration should commence at a dose of 100 mg twice per day.
- In patients with moderate renal impairment (adjusted creatinine clearance of 30-50 mL/min/1.73m2), ZAVESCA administration should commence at a dose of one 100 mg capsule per day.
- Use of ZAVESCA in patients with severe renal impairment (creatinine clearance < 30 mL/min/1.73 m2) is not recommended.
IMPORTANT SAFETY INFORMATION
- ZAVESCA is contraindicated in patients who have demonstrated hypersensitivity to the active substance or any of the excipients.
Pregnancy Category X
- ZAVESCA may cause fetal harm when administered to a pregnant woman.
- ZAVESCA is contraindicated in women who are or may become pregnant. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.
WARNINGS AND PRECAUTIONS
Peripheral neuropathy has been reported in patients treated with ZAVESCA.
- Patients should undergo neurological examination at the start of treatment and every 6 months thereafter.
- Patients who develop symptoms such as numbness and tingling should have a careful re-assessment of the risk/benefit of ZAVESCA therapy and cessation of treatment may be considered.
Approximately 30% of patients taking ZAVESCA have reported tremor or exacerbation of existing tremor.
- These tremors were described as an exaggerated physiological tremor of the hands.
- Tremor usually began within the first month of therapy and in many cases resolved within 1 to 3 months.
- Dose reduction may ameliorate the tremor, usually within days, but discontinuation of treatment may sometimes be required.
Diarrhea and Weight Loss
- Diarrhea and weight loss were common in clinical studies of patients treated with ZAVESCA, with approximately 85% and up to 65% of treated patients, respectively, reporting these conditions.
- The incidence of diarrhea was noted to decrease over time with continued ZAVESCA treatment, and was observed to respond to individualized diet modification, to taking ZAVESCA between meals, and/or to anti-diarrheal medications, most commonly loperamide.
Reductions in Platelet Count
- Mild reductions in platelet counts without association with bleeding were observed in some patients with another disease treated with ZAVESCA in a clinical trial.
- Male patients should maintain reliable contraceptive methods while taking ZAVESCA.
- Studies in the rat have shown that miglustat adversely affects spermatogenesis and sperm parameters, thereby reducing fertility.
- Until further information is available, it is advised that before seeking to father a child, male patients should cease ZAVESCA and maintain reliable contraceptive methods for 3 months thereafter.
- The most common serious adverse reaction reported with ZAVESCA treatment in clinical studies was peripheral neuropathy.
- The most common treatment-emergent adverse events reported in clinical studies with ZAVESCA were weight loss, diarrhea, and tremor.
- Other common adverse reactions were*:
- Tremor (11% to 17%)
- Diarrhea (89% to 100%)
- Weight Decrease (39% to 67%)
- Thrombocytopenia (0% to 7%)
- Abdominal Pain (18% to 67%)
- Flatulence (29% to 50%)
- Nausea (8% to 22%)
- Vomiting (4% to 11%)
- Headache (0% to 22%)
- Cramps Legs (4% to 11%)
- Dizziness (0% to 11%)
- Weakness Generalized (0% to 17%)
- Visual Disturbance (0% to 17%)
- Abdominal Distension/Bloating (0% to 8%)
- Back Pain (0% to 8%)
- Mouth Dry (0% to 8%)
- Constipation (0% to 8%)
- Heaviness in Limbs (0% to 8%)
- Gait Unsteady (0% to 8%)
- Memory Loss (0% to 8%)
- Anorexia (0% to 7%)
- Dyspepsia (0% to 7%)
- Mental Disorder (0% to 6%)
||Adverse Events reported above exclude those reported in the combination arm of Study 3, section 6.1 of the PI.
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