Miglustat Gen.Orph

Land: Den Europæiske Union

Sprog: engelsk

Kilde: EMA (European Medicines Agency)

Aktiv bestanddel:

miglustat

Tilgængelig fra:

Gen.Orph

ATC-kode:

A16AX06

INN (International Name):

miglustat

Terapeutisk gruppe:

Other alimentary tract and metabolism products,

Terapeutisk område:

Gaucher Disease

Terapeutiske indikationer:

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.

Produkt oversigt:

Revision: 9

Autorisation status:

Authorised

Autorisation dato:

2017-11-09

Indlægsseddel

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

Produktets egenskaber

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