Страна: Европейски съюз
Език: английски
Източник: EMA (European Medicines Agency)
miglustat
Gen.Orph
A16AX06
miglustat
Other alimentary tract and metabolism products,
Gaucher Disease
Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
Revision: 9
Authorised
2017-11-09
19 B. PACKAGE LEAFLET 20 PACKAGE LEAFLET: INFORMATION FOR THE USER MIGLUSTAT GEN.ORPH 100 MG HARD CAPSULES miglustat READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START TAKING THIS MEDICINE BECAUSE IT CONTAINS IMPORTANT INFORMATION FOR YOU. - Keep this leaflet. You may need to read it again. - If you have any further questions, ask your doctor or pharmacist. - This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours. - If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4. WHAT IS IN THIS LEAFLET 1. What Miglustat Gen.Orph is and what it is used for 2. What you need to know before you take Miglustat Gen.Orph 3. How to take Miglustat Gen.Orph 4. Possible side effects 5. How to store Miglustat Gen.Orph 6. Contents of the pack and other information 1. WHAT MIGLUSTAT GEN.ORPH IS AND WHAT IT IS USED FOR Miglustat Gen.Orph contains the active substance miglustat which belongs to a group of medicines that affect metabolism. It is used to treat two conditions: • MIGLUSTAT GEN.ORPH IS USED TO TREAT MILD TO MODERATE TYPE 1 GAUCHER DISEASE IN ADULTS. In type 1 Gaucher disease, a substance called glucosylceramide is not removed from your body. It starts to build up in certain cells of the body’s immune system. This can result in liver and spleen enlargement, changes in the blood and bone disease. The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Miglustat Gen.Orph is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy. • MIGLUSTAT GEN.ORPH IS ALSO USED TO TREAT PROGRESSIVE NEUROLOGICAL SYMPTOMS IN NIEMANN- PICK TYPE C DISEASE IN ADULTS AND IN CHILDREN. If you have Niemann-Pick type C disease, fats such as glycosphingolipids build up in the cells of your brain. This can result in disturbances in neurological functions such as slow eye movements, balance, swallowing, and Прочетете целия документ
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 1. NAME OF THE MEDICINAL PRODUCT Miglustat Gen.Orph 100 mg hard capsules 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each hard capsule contains 100 mg miglustat For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Hard capsule. White opaque cap and body, hard gelatin capsules size 4 of about 14 mm length. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1). Miglustat Gen.Orph 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). 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate. Posology _Dose in type 1 Gaucher disease _ _ _ _Adult _ 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. _ _ _Paediatric population _ The efficacy of miglustat in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. No data are available. _Dose in Niemann-Pick type C disease _ _Adult _ The recommended dose for the treatment of adult patients with Niemann-Pick type C disease is 200 mg three times a day. 3 _Paediatric population _ 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: TABLE 1. – PAEDIATRIC POPULATION Body s Прочетете целия документ