Yargesa

Maa: Euroopan unioni

Kieli: englanti

Lähde: EMA (European Medicines Agency)

Aktiivinen ainesosa:

miglustat

Saatavilla:

Piramal Critical Care B.V.

ATC-koodi:

A16AX06

INN (Kansainvälinen yleisnimi):

miglustat

Terapeuttinen ryhmä:

Other alimentary tract and metabolism products,

Terapeuttinen alue:

Gaucher Disease

Käyttöaiheet:

Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

Tuoteyhteenveto:

Revision: 5

Valtuutuksen tilan:

Authorised

Valtuutus päivämäärä:

2017-03-22

Pakkausseloste

19
B.
PACKAGE LEAFLET
20
PACKAGE LEAFLET: INFORMATION FOR THE USER
YARGESA 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 Yargesa is and what it is used for
2.
What you need to know before you take Yargesa
3.
How to take Yargesa
4.
Possible side effects
5.
How to store Yargesa
6.
Contents of the pack and other information
1.
WHAT YARGESA IS AND WHAT IT IS USED FOR
Yargesa contains the active substance miglustat which belongs to a
group of medicines that affect
metabolism. It is used to treat two conditions:
•
YARGESA 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. Yargesa is only used
when a patient is considered unsuitable for treatment with enzyme
replacement therapy.
•
YARGESA 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 memory, and in seizures.
Yargesa works by inhibiting the enzyme called ‘glucosylceramide
synthase’ which i

Valmisteyhteenveto

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
1.
NAME OF THE MEDICINAL PRODUCT
Yargesa 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.
The hard capsule consists of an opaque white cap and body with
“708” printed in black on the body.
Capsule size: 4 (14.3 mm x 5.3 mm)
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Yargesa is indicated for the oral treatment of adult patients with
mild to moderate type 1 Gaucher
disease.
Yargesa may be used only in the treatment of patients for whom enzyme
replacement therapy is
unsuitable(see sections 4.4 and 5.1).
Yargesa 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 t_
_ype 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 N_
_iemann-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.
_Paediatric population _
3
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 (