Miglustat Gen.Orph
Italy
Package leaflet: Information for the user
Miglustat Gen.Orph 100 mg hard capsules
miglustat
Read this leaflet carefully before 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 give it to other people, even if their symptoms are the same as yours, as it may be harmful. If you experience any side effects, including those not listed in this leaflet, contact your doctor or pharmacist. See section 4.
Contents of this leaflet:
- What Miglustat Gen.Orph is and what it is used for.
- What you need to know before taking Miglustat Gen.Orph.
- How to take Miglustat Gen.Orph.
- Possible side effects.
- How to store Miglustat Gen.Orph.
- 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 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 properly eliminated from your
body. It accumulates in certain cells of the immune system. This accumulation can lead to enlargement
of the liver and spleen, blood cell abnormalities, and bone disease.
The standard treatment for type 1 Gaucher disease is 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 disease type C.
If you have Niemann-Pick disease type C, fats (such as glycosphingolipids) accumulate within brain
cells. This can cause neurological problems such as slow eye movements, imbalance, difficulty swallowing,
memory problems, and seizures.
Miglustat Gen.Orph works by inhibiting an enzyme called "glucosylceramide synthase," which is
responsible for the first step in 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 to 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 have kidney problems;
- if you have liver problems.
Before and during treatment with Miglustat Gen.Orph, your doctor will carry out the following checks:
- examination to monitor the condition of the nerves in your arms and legs;
- measurement of vitamin B\ levels;
- monitoring of growth, in pediatric or adolescent patients with Niemann-Pick disease type C;
- monitoring of blood platelet count.
The reason these checks are performed is that, during treatment with Miglustat Gen.Orph,
some patients have experienced tingling or numbness in the hands and feet, or a decrease in body
weight. These checks will help your doctor determine whether such effects are due to your disease or
other existing conditions, or are side 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 carbohydrate intake such as sucrose (cane sugar); decide not to take Miglustat Gen.Orph with food; or temporarily reduce the dose of Miglustat Gen.Orph. In some cases, your doctor may prescribe antidiarrhoeal medicines such as loperamide. If diarrhoea does not respond to the above measures or if you have any other abdominal symptoms, consult your doctor. In such cases, your doctor may decide to perform further investigations.
Male patients must use reliable contraceptive methods during treatment with Miglustat Gen.Orph and for 3 months after treatment has ended.
Children and adolescents
Do not administer this medicine to children and adolescents (under 18 years of age) with type 1 Gaucher disease, as it is not known whether the medicine is effective in this condition.
Other medicines and Miglustat Gen.Orph
Inform your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Inform your doctor if you are taking other medicines containing imiglucerase, which are sometimes used concurrently with Miglustat Gen.Orph. These may reduce your body's exposure to Miglustat Gen.Orph.
Pregnancy, breastfeeding and fertility
If you are pregnant, think you might be pregnant, or are planning to become pregnant, you must not take Miglustat Gen.Orph. Your doctor can provide you with further information. You must use an effective method of contraception while taking Miglustat Gen.Orph. Do not breastfeed while taking Miglustat Gen.Orph.
Male patients must continue to use reliable contraceptive methods during treatment with Miglustat Gen.Orph and for 3 months after treatment has ended.
If you are pregnant, think you might be pregnant, are planning to become pregnant, or are breastfeeding, ask your doctor or pharmacist for advice before taking this medicine.
Driving and using machines
Miglustat Gen.Orph may cause dizziness. If you experience dizziness, do not drive 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 exactly as your doctor has told you. If you are unsure, consult your doctor or pharmacist.
- For type 1 Gaucher disease: the usual dose for adults is one capsule (100 mg) three times daily (morning, afternoon, and evening), for a maximum of three capsules per day (300 mg).
- For Niemann-Pick disease type C: the usual dose for adults and adolescents (over 12 years of age) is two capsules (200 mg) three times daily (morning, afternoon, and evening), corresponding to a maximum daily dose of six capsules (600 mg).
For children under 12 years of age, your doctor will appropriately adjust the dose to be used for Niemann-Pick disease type C.
If you have kidney problems, you may initially receive a lower dose. Your doctor may decide to reduce the dose; for example, one capsule (100 mg) once or twice daily, especially if you experience diarrhoea while taking Miglustat Gen.Orph (see section 4). Your doctor will decide and inform you on 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 prescribed, contact your doctor immediately. In clinical studies, miglustat has been used at doses up to 3000 mg, which caused reductions in white blood cell count and other adverse effects similar to those described in section 4.
If you forget to take Miglustat Gen.Orph
Take the next capsule at your usual time. Do not take a double dose to make up for the missed dose.
If you stop taking Miglustat Gen.Orph
Do not stop taking Miglustat Gen.Orph without first talking to your doctor.
If you have any questions about how to use 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, caused by side effects of Miglustat Gen.Orph, or could be due to pre-existing conditions. To assess them, your doctor will carry out certain 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 slight tremor, usually hand tremors, seek advice from your doctor as soon as possible.
The tremor often disappears without the need to stop treatment. To resolve the tremor, your doctor may sometimes need to reduce the dose or stop treatment with Miglustat Gen.Orph.
Very common side effects (may affect more than 1 in 10 people)
The most common side effects include: diarrhoea, flatulence (intestinal gas), abdominal pain (stomach), weight loss and reduced appetite.
If you lose weight when starting treatment with Miglustat Gen.Orph, do not worry. Weight loss usually stops as treatment continues.
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 (indigestion), nausea (feeling sick), constipation and vomiting, abdominal swelling or discomfort (stomach), thrombocytopenia (reduced platelet levels in the blood).
Neurological symptoms and thrombocytopenia may be due to the underlying disease.
Other possible side effects include: weakness or muscle spasms, fatigue, chills and malaise, depression, difficulty sleeping, memory loss and decreased libido.
Most patients report one or more of these side effects, usually at the beginning of treatment or intermittently during treatment. These cases are mostly mild and disappear quickly. If any of these side effects cause problems, consult your doctor. They may reduce your dose of Miglustat Gen.Orph or recommend other medicines to counteract the side effects.
Reporting of side effects
If you get any side effects, including ones not listed in this leaflet, talk to your doctor or pharmacist. You can also report side effects directly through the national reporting system detailed 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 stated on the carton after the word "Exp.". The expiry date refers to the last day of that month.
This medicine does not require any special storage conditions.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. This will help protect the environment.
6. Package contents and other information
What Miglustat Gen.Orph contains
- The active substance 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 package contents
Miglustat Gen.Orph 100 mg hard capsules have an opaque white cap and body, hard gelatin capsules of size 4, with a length of 14 mm.
Pack sizes: 84 hard capsules in non-perforated blisters and 84x1 hard capsules in unit-dose divisible blisters.
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 about this medicinal product, please contact the local representative of the Marketing Authorisation Holder.
België/Belgique/Belgien Lietuva
Gen.Orph Gen.Orph
Tél/Tel: +32 (0)496 85 87 49 Tel: +33 (0)1 47 71 04 50
e-mail: [email protected] e-mail: [email protected]
България Luxembourg/Luxemburg
Диакомерс ЕООД Gen.Orph
Тел.: +359 2 807 50 00 Tél/Tel.: +32 (0)496 85 87 49
Е-mail: [email protected] email: [email protected]
Česká republika Magyarország
Gen.Orph HMS Pharma Kft.
Tel: +33 (0)1 47 71 04 50 Tel.: +36 30 375 8124
e-mail: [email protected] e-mail: [email protected]
Danmark Malta
Gen.Orph Gen.Orph
Tlf: +46 (0)8 21 54 45 Tel: +33 (0)1 47 71 04 50
e-mail: [email protected]
Deutschland Nederland
Gen.Orph Gen.Orph
Tel: +49 30 8560687897 Tel: +32 (0)496 85 87 49
email: [email protected]
Eesti Norge
Gen.Orph Gen.Orph
Tel: +33 (0)1 47 71 04 50 Tlf: +46 (0)8 21 54 45
email: [email protected] e-mail: [email protected]
Ελλάδα Österreich
Gen.Orph Gen.Orph
Τηλ: +33 (0)1 47 71 04 50 Tel: +33 (0)1 47 71 04 50
email: [email protected] e-mail: [email protected]
España Polska
Pharma International S.A. Gen.Orph
Tel: +34 915 635 856 Tel.: +33 (0)1 47 71 04 50
e-mail: [email protected] e-mail: [email protected]
France Portugal
Gen.Orph Biojam, S.A.
Tél.: +33 (0)1 47 71 04 50 Tel: +351 212 697 912
e-mail: [email protected] e-mail: [email protected]
Hrvatska România
Gen.Orph Gen.Orph
Tel: +33 (0)1 47 71 04 50 Tel: +33 (0)1 47 71 04 50
e-mail: [email protected] e-mail: [email protected]
Ireland Slovenija
Gen.Orph Gen.Orph
Tel: +33 (0)1 47 71 04 50 Tel: +33 (0)1 47 71 04 50
email: [email protected] email: [email protected]
Ísland Slovenská republika
Gen.Orph Gen.Orph
Sími: +33 (0)1 47 71 04 50 Tel: +33 (0)1 47 71 04 50
e-mail: [email protected] e-mail: [email protected]
Italia Suomi/Finland
Biovalley Investments Partner S.r.l. Gen.Orph
Tel: +39 040 899 2219 Puh/Tel: +46 (0)8 21 54 45
e.mail: [email protected] e-mail: [email protected]
Κύπρος Sverige
Gen.Orph Gen.Orph
Τηλ: +33 (0)1 47 71 04 50 Tel: +46 (0)8 21 54 45
email: [email protected] e-mail: [email protected]
Latvija United Kingdom
Gen.Orph Gen.Orph
Tel: +33 (0)1 47 71 04 50 Tel: +33 (0)1 47 71 04 50
e-mail: [email protected] 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 their therapeutic treatments are provided.