Source: European Medicines Agency (EU) Revision Year: 2022 Publisher: Janssen-Cilag International NV, Turnhoutseweg 30, B-2340, Beerse, Belgium
Zavesca is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1).
Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (see sections 4.4, and 5.1).
Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate.
The recommended starting dose for the treatment of adult patients with type 1 Gaucher disease is 100 mg three times a day.
Temporary dose reduction to 100 mg once or twice a day may be necessary in some patients because of diarrhoea.
The efficacy of Zavesca in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. No data are available.
The recommended dose for the treatment of adult patients with Niemann-Pick type C disease is 200 mg three times a day.
The recommended dose for the treatment of adolescent patients (12 years of age and above) with Niemann-Pick type C disease is 200 mg three times a day.
Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below:
| Body surface area (m²) | Recommended dose |
|---|---|
| >1.25 | 200 mg three times a day |
| >0.88-1.25 | 200 mg twice a day |
| >0.73-0.88 | 100 mg three times a day |
| >0.47-0.73 | 100 mg twice a day |
| ≤0.47 | 100 mg once a day |
Temporary dose reduction may be necessary in some patients because of diarrhoea.
The benefit to the patient of treatment with Zavesca should be evaluated on a regular basis (see section 4.4).
There is limited experience with the use of Zavesca in Niemann-Pick type C disease patients under the age of 4 years.
There is no experience with the use of Zavesca in patients over the age of 70.
Pharmacokinetic data indicate increased systemic exposure to miglustat in patients with renal impairment. In patients with an adjusted creatinine clearance of 50–70 ml/min/1.73 m², administration should commence at a dose of 100 mg twice daily in patients with type 1 Gaucher disease and at a dose of 200 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
In patients with an adjusted creatinine clearance of 30–50 ml/min/1.73 m², administration should commence at a dose of 100 mg once daily in patients with type 1 Gaucher disease and at a dose of 100 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease. Use in patients with severe renal impairment (creatinine clearance <30 ml/min/1.73 m²) is not recommended (see sections 4.4 and 5.2).
Zavesca has not been evaluated in patients with hepatic impairment.
Zavesca can be taken with or without food.
No acute symptoms of overdose have been identified. Zavesca has been administered at doses of up to 3000 mg/day for up to six months in HIV positive patients during clinical trials. Adverse events observed included granulocytopenia, dizziness and paraesthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or higher dose.
In case of overdose general medical care is recommended.
5 years.
Do not store above 30°C.
ACLAR/ALU blister strips supplied as a box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules.
No special requirements for disposal.
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