MIGLUSTAT 100 mg Hard Capsule

Содержание инструкции

1. Miglustat Gen.Orph 100 mg hard capsules
   1. What is Miglustat Gen.Orph and what is it used for
   2. What you need to know before taking 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

Miglustat Gen.Orph 100 mg hard capsules

miglustat

Read 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 questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you only. Do not pass it on to others, even if their symptoms are the same as yours, as it could be harmful. If you get any side effects, even those not listed in this leaflet, tell your doctor or pharmacist. See section 4.

Contents of 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 is Miglustat Gen.Orph and what is it used for

Miglustat Gen.Orph contains the active ingredient miglustat, which belongs to a group of medicines that
act on metabolism. It is used to treat two diseases:

• 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 eliminated from the
body. It accumulates in some cells of the immune system. This accumulation can lead to
enlargement of the liver and spleen, alterations of blood cells and bone disease.
The usual treatment for type 1 Gaucher disease consists of enzyme replacement therapy. Miglustat Gen.Orph is used only if a patient is considered
unsuitable for treatment with enzyme replacement therapy.

• Miglustat Gen.Orph is also used, in adults and children, to treat progressive neurological symptoms in Niemann-Pick type C disease

If you have Niemann-Pick type C disease, fats (such as, for example, glycosphingolipids)
increase within brain cells. This can cause disturbances in
neurological functions such as slow eye movements, balance, swallowing, memory and seizures.
Miglustat Gen.Orph works by inhibiting an enzyme called “glucosylceramide synthase” which is
responsible for the first stage of the synthesis of most glycosphingolipids.

2. What you need to know before taking Miglustat Gen.Orph

Do not take Miglustat Gen.Orph:

• if you are allergic to miglustat or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions
Talk to your doctor or pharmacist before taking Miglustat Gen.Orph :

• if you suffer from kidney problems;
• if you suffer from liver problems.

Before and during treatment with Miglustat Gen.Orph, your doctor will perform the following checks:

• an examination to check the condition of the nerves in your arms and legs
• measurement of vitamin B levels
• monitoring of growth, in the case of a paediatric or adolescent patient with Niemann-Pick disease type C
• checking the number of platelets in your blood

The reason these checks are performed is that, during the intake of Miglustat Gen.Orph,
some patients have experienced tingling or numbness in the hands and feet, or a decrease in body
weight. The checks will help your doctor determine if these effects are due to your illness or to
other existing conditions or are undesirable effects of Miglustat Gen.Orph (for further information
see section 4).
If you have diarrhoea, your doctor may decide to modify your diet to reduce lactose and the consumption
of carbohydrates such as sucrose (cane sugar); decide not to have you take Miglustat
Gen.Orph with food; decide to temporarily reduce the dose of Miglustat Gen.Orph. In
some cases your doctor may prescribe anti-diarrhoeal medicines such as loperamide. If the
diarrhoea does not respond to the above measures or if you have any other abdominal discomfort, consult
your doctor. In this case, your doctor may decide to carry out further investigations.
Male patients, during treatment with Miglustat Gen.Orph and for 3 months after its conclusion,
must use reliable contraceptive methods.

Children and adolescents
Do not give this medicine to children and adolescents (under 18 years of age) with type 1 Gaucher’s disease, as it is not known whether the medicine works in this disease.

Other medicines and Miglustat Gen.Orph
Tell your doctor or pharmacist if you are taking, have recently taken or might take
any other medicines.
Tell your doctor if you are taking other medicines containing imiglucerase, which are sometimes used
at the same time as Miglustat Gen.Orph. They may decrease your body's exposure to
Miglustat Gen.Orph.

Pregnancy, breastfeeding and fertility
If you are pregnant, suspect you are pregnant or are planning to become pregnant, you should not take
Miglustat Gen.Orph. Your doctor will be able to provide you with further information. During the intake of
Miglustat Gen.Orph you must use an effective method of contraception. Do not breastfeed while
taking Miglustat Gen.Orph.
Male patients, during the intake of Miglustat Gen.Orph, and for 3 months after the end of
treatment, must continue to use reliable contraceptive methods.
If you are pregnant, suspect you are pregnant or are planning to become pregnant or if you are breastfeeding
with breast milk, ask your doctor or pharmacist for advice before taking this medicine.

Driving and operating machinery
Miglustat Gen.Orph can cause dizziness. If you feel dizzy, do not drive vehicles or operate
tools or machinery.

Miglustat Gen.Orph contains sodium
This medicine contains less than 1 mmol (23 mg) of sodium per hard capsule, i.e. essentially
"sodium-free".

3. How to take Miglustat Gen.Orph

Take this medicine following exactly the instructions of your doctor. If you have any doubts, ask your doctor or pharmacist.

• For Gaucher's disease type 1: for adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon and evening), equivalent to a maximum of three capsules a day (300 mg).
• For Niemann-Pick disease type C: for adults and adolescents (over 12 years of age) the usual dose is two capsules (200 mg) 3 times a day (morning, afternoon and evening). This means a maximum daily dose of six capsules (600 mg).

For children under 12 years of age, the doctor will appropriately adjust the dose of medication to be used for Niemann-Pick disease type C.
If you have kidney problems, at the beginning of treatment, you may receive a lower dose. Your doctor may decide to reduce the dose; for example, one capsule (100 mg), one or two times a day, if you suffer from diarrhoea while taking Miglustat Gen.Orph (see section 4). The doctor will decide and communicate how long the treatment should last.
Miglustat Gen.Orph can be taken on an empty stomach or with food. The capsule must be swallowed whole with a glass of water.

If you take more Miglustat Gen.Orph than you should
If you take more capsules than you should, consult your doctor immediately. In clinical studies, miglustat has been used at doses up to 3000 mg: this has caused decreases in the number of white blood cells and other undesirable effects, similar to those described in section 4.

If you forget to take Miglustat Gen.Orph
Take the next capsule at the usual time. Do not take a double dose to make up for a forgotten dose.

If you stop taking Miglustat Gen.Orph
Do not stop taking Miglustat Gen.Orph without talking to your doctor.
If you have any further questions on the use of this medicine, ask your doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
Serious side effects

Some patients have experienced tingling or numbness in the hands and feet (commonly observed).These could be signs of peripheral neuropathy, due to side effects of
Miglustat Gen.Orph or they may be due to pre-existing conditions. To evaluate them, the doctor
will perform some checks before and during treatment with Miglustat Gen.Orph (see section 2).

If you experience any of these effects, please seek medical advice from your doctor
as soon as possible.
If you experience a mild tremor, usuallytremor in the hands, seek advice from your
doctoras soon as possible. The tremor often disappears without the need to interrupt the
treatment. To make the tremor disappear, your doctor may sometimes need to reduce the dose or
interrupt treatment with Miglustat Gen.Orph.

Very common side effects(may affect more than 1 in 10 people)
The most common side effects include: diarrhea, flatulence (intestinal gas), abdominal
pain (stomach), weight loss and decreased appetite.

If you lose weightwhen you start treatment with Miglustat Gen.Orph, do not worry. Weight loss
usually stops as treatment progresses.

Common side effects(may affect up to 1 in 10 people)
Common side effects of treatment include headache, dizziness, paresthesia (tingling or
numbness), abnormal coordination, hypoesthesia (reduced sensitivity to touch), dyspepsia (heartburn),
nausea (feeling sick), constipation and vomiting, abdominal (stomach) swelling or discomfort,
thrombocytopenia (reduced levels of platelets in the blood).
Neurological symptoms and thrombocytopenia may be due to the underlying disease.

Other possible side effectsare: muscle weakness or spasms, fatigue, chills and
malaise, depression, difficulty sleeping, memory loss and decreased libido.
Most patients complain of one or more of these side effects, usually at the beginning of
treatment or at intervals during treatment. These cases are mostly mild and disappear
quickly. If any of these side effects cause problems, consult your doctor. They
may reduce the dose of Miglustat Gen.Orph or recommend other medicines to counteract the
side effects.

Reporting of side effects
If you get any side effects, even if not listed in this leaflet, tell your doctor or pharmacist. You can also
report side effects directly through the national reporting system listed in Annex V. By reporting side
effects you can help provide more information on the safety of this medicine.

5. How to store Miglustat Gen.Orph

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the pack after the words
“Exp.”. The expiry date refers to the last day of that month.
This medicine does not require any particular storage conditions.
Do not dispose of any medicine in waste water or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Contents of the pack and other information

What Miglustat Gen.Orph contains

• The active ingredient is miglustat 100 mg.
• The other ingredients are sodium starch glycolate (type A), povidone (K30), magnesium stearate, gelatin, titanium dioxide (E171). See section 2 "Miglustat Gen.Orph contains sodium".

Description of the appearance of Miglustat Gen.Orph and contents of the pack
The hard capsules of Miglustat Gen.Orph 100 mg have opaque white cap and body, hard
gelatin capsules of size 4, with a length of 14 mm.
Pack of 84 hard capsules in non-perforated blisters and 84x1 hard capsules in divisible blisters for
unit dose.
Not all pack sizes may be marketed.

Marketing authorisation holder and manufacturer
Gen.Orph
185 Bureaux de la Colline
92213 Saint Cloud Cedex
France

Manufacturers
Delpharm Reims
10 rue Colonel Charbonneaux
51100 Reims
France
or
Centre Lab
ZA Granderaie
23000 Guéret
France
For further information on this medicinal product, contact the local representative of the marketing
authorisation holder.

België/Belgique/Belgien
Gen.Orph
Tél/Tel: +32 (0)496 85 87 49
e-mail: [email protected]

Lietuva
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

България
Диакомерс ЕООД
Тел.: +359 2 807 50 00
Е-mail: [email protected]

Luxembourg/Luxemburg
Gen.Orph
Tél/Tel.: +32 (0)496 85 87 49
email: [email protected]

Česká republika
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

Magyarország
HMS Pharma Kft.
Tel.: +36 30 375 8124
e-mail: [email protected]

Danmark
Gen.Orph
Tlf: +46 (0)8 21 54 45
e-mail:

Malta
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail : [email protected]

Deutschland
Gen.Orph
Tel: +49 30 8560687897
email:

Nederland
Gen.Orph
Tel: +32 (0)496 85 87 49
e-mail: [email protected]

Eesti
Gen.Orph
Tel: +33 (0)1 47 71 04 50
email: [email protected]

Norge
Gen.Orph
Tlf: +46 (0)8 21 54 45
e-mail : [email protected]

Ελλάδα
Gen.Orph
Τηλ: +33 (0)1 47 71 04 50
email: [email protected]

Österreich
Gen.Orph
Tel : +33 (0)1 47 71 04 50
e-mail: [email protected]

España
Pharma International S.A.
Tel: +34 915 635 856
e-mail: [email protected]

Polska
Gen.Orph
Tel.: +33 (0)1 47 71 04 50
e-mail: [email protected]

France
Gen.Orph
Tél.: +33 (0)1 47 71 04 50
e-mail : [email protected]

Portugal
Biojam, S.A.
Tel: +351 212 697 912
e-mail: [email protected]

Hrvatska
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

România
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

Ireland
Gen.Orph
Tel : +33 (0)1 47 71 04 50
email: [email protected]

Slovenija
Gen.Orph
Tel: +33 (0)1 47 71 04 50
email: [email protected]

Ísland
Gen.Orph
Sími: +33 (0)1 47 71 04 50
e-mail : [email protected]

Slovenská republika
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

Italia
Biovalley Investments Partner S.r.l.
Tel: +39 040 899 2219
e.mail: [email protected]

Suomi/Finland
Gen.Orph
Puh/Tel : +46 (0)8 21 54 45
e-mail : [email protected]

Κύπρος
Gen.Orph
Τηλ: +33 (0)1 47 71 04 50
email: [email protected]

Sverige
Gen.Orph
Tel: +46 (0)8 21 54 45
e-mail: [email protected]

Latvija
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]

United Kingdom
Gen.Orph
Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]
More detailed information on this medicinal product is available on the website of the European Medicines
Agency: http://www.ema.europa.eu. In addition, links to other websites on rare diseases and related
therapeutic treatments are provided.