Zavesca 100 mg hard capsules

Zavesca belongs to a group of medicines that affect metabolism. It is used to treat two conditions:

• Zavesca is used to treat mild to moderate type 1 Gaucher disease.

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. Zavesca is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy.

• Zavesca is also used to treat progressive neurological symptoms in Niemann-Pick type C disease.

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 eye movements, balance, swallowing, and memory, and in seizures.

Zavesca works by inhibiting the enzyme called ?glucosylceramide synthase? which is responsible for the first step in the synthesis of most glycosphingolipids.

Do not take Zavesca

• if you are allergic (hypersensitive) to miglustat or any of the other ingredients of Zavesca. If this applies to you, tell your doctor. Don?t take Zavesca.

Take special care with Zavesca

• if you suffer from kidney disease
• if you suffer from liver disease If any of these apply to you, tell your doctor before you take Zavesca.

Your doctor will perform the following tests before treatment and during treatment with Zavesca:

• an examination to check the nerves in your arms and legs - measurement of vitamin B 12 levels
• monitoring growth if you are a child or adolescent with Niemann-Pick type C disease
• monitoring of blood platelet counts if you are a patient with Niemann-Pick type C disease

The reason for these tests is that some patients have had tingling or numbness in the hands and feet, or a decrease in body weight, while taking Zavesca. The tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Zavesca (see section 4 for further details).

If you have diarrhoea, your doctor may ask you to change your diet to reduce your lactose and carbohydrate intake, or not to take Zavesca together with food, or to temporarily reduce your dose. In some cases the doctor may prescribe anti-diarrhoeal medicines such as loperamide. If your diarrhoea does not respond to these measures, or if you have any other abdominal complaint, consult your doctor. In such case, your doctor may decide to conduct further investigations.

Male patients should use reliable birth control methods during their treatment with Zavesca, and for 3 months after finishing treatment.

Using other medicines

Talk to your doctor if you are using Cerezyme, or other medicines containing imiglucerase, which are sometimes used at the same time as Zavesca. They may lower the amount of Zavesca in your body. Please tell your doctor or pharmacist if you are using, or have recently used, any other medicines, including medicines obtained without prescription.

Taking Zavesca with food and drink
Zavesca can be taken with or without food. You should swallow the whole capsule with a glass of water.

Pregnancy and breast-feeding
You should not use Zavesca if you are pregnant or thinking of becoming pregnant. Your doctor can give you more information. You must use effective birth control while using Zavesca. Do not breast-feed while you are using Zavesca.

Male patients should use reliable birth control methods during their treatment with Zavesca, and for 3 months after finishing treatment.

Ask your doctor or pharmacist for advice before taking any medicine.

Driving and using machines
Zavesca may make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy.

Always take Zavesca exactly as your doctor has told you. You should check with your doctor or pharmacist if you are not sure about any of the instructions.

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

If you are less than 12 years old, your doctor will adjust your dose for Niemann-Pick type C disease.

If you have a problem with your kidneys you may receive a lower starting dose. Your doctor may reduce your dose, e.g., to one capsule (100 mg) once or twice a day, if you suffer from diarrhoea when taking Zavesca (see section 4). Your doctor will tell you how long your treatment will last.

To remove the capsule:

1. Separate at perforations

2. Peel back paper at arrows

3. Push product through foil

The Zavesca capsule should be swallowed whole with water.

If you take more Zavesca than you should

If you take more capsules than you were told to, consult your doctor immediately. Zavesca has been used in clinical trials at doses ten times higher than the recommended dose: this caused decreases in white blood cells and other side effects similar to those described in section 4.

If you forget to take Zavesca
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 Zavesca

Don?t stop taking Zavesca without talking to your doctor.

If you have any further questions on the use of this medicine, ask your doctor or pharmacist.

Like all medicines, Zavesca can cause side effects, although not everybody gets them.

Very common effects ? likely to affect more than 1 in 10 people
The most common side effects are weight loss, tremor, diarrhoea, flatulence (wind), abdominal (stomach) pain.

Common effects ?likely to affect less than 1 in 10 people and more than 1 in 100 people Common side effects of treatment include anorexia(lack of appetite), decreased appetite, headache, dizziness, peripheral neuropathy, paraesthesia (tingling or numbness), abnormal coordination, hypoaesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling sick), constipation and vomiting, swelling or discomfort in the abdomen (stomach) and thrombocytopenia (reduced levels of blood platelets).The neurological symptoms and thrombocytopenia could be due to the underlying disease.

Other possible side effects are muscular spasms or weakness , fatigue, difficulty sleeping , and less libido.

Most patients get one or more of these side effects, usually at the start of treatment or at intervals during treatment. Most cases are mild and disappear quite quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of Zavesca or recommend other medicines to help control side effects.

Some patientshave had tingling or numbness in the hands and feet. They could be signs of peripheral neuropathy, due to side effects of Zavesca or they could be due to existing conditions. Your doctor will perform some tests before and during treatment with Zavesca to assess this (see section 2). If you do get any of these effects, please report them to your doctor as soon as possible.

If you do lose some weight when you start treatment with Zavesca don?t worry. People usually stop losing weight as treatment goes on.

If youget a slight tremor, usually trembling hands, report it to your doctor as soon as possible. The tremor often disappears without needing to stop the treatment. Sometimes your doctor will need to reduce the dose or stop Zavesca treatment to stop the tremor.

If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor or pharmacist.

For storage and other information, please turn over.

Keep out of the reach and sight of children.

Do not use Zavesca after the expiry date stated on the carton.

Store below 30 °C.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.

What Zavesca contains

The active substance is miglustat 100 mg.

Other ingredients in the capsule are:

Sodium starch glycollate,
Povidone (K30),
Magnesium stearate.

Other ingredients in the capsule shell are:

Gelatin,
Water,
Titanium dioxide (E171).

Other ingredients in the Printing ink are:

Black iron oxide (E172)
Shellac.

What Zavesca looks like and contents of the pack

Zavesca is a white 100 mg capsule with ?OGT 918? printed in black on the cap and ?100? printed in black on the body.
Box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules.

Marketing Authorisation Holder:

Actelion Registration Ltd
BSI Building 13 th Floor
389 Chiswick High Road
London W4 4AL
United Kingdom

Manufacturer responsible for batch release:

Actelion Pharmaceuticals Deutschland GmbH
Basler Strasse 63?65
79100 Freiburg
Germany

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder.

BelgiëBelgiqueBelgien Actelion Pharmaceuticals Belgium N.V. TélTel 32-015 284 777 LuxembourgLuxemburg Actelion Pharmaceuticals Belgium N.V. TélTel 32-015 284 777

Actelion Pharmaceuticals Ltd Te. 420-2 3413 8150 Magyarország Actelion Pharmaceuticals Ltd Tel 48 0 500 145 920

eská republika Actelion Pharmaceuticals CZ, s.r.o. Tel 420 2 21 968 006 Malta Actelion Pharmaceuticals Ltd Tel 48 0 500 145 920

Danmark Actelion Pharmaceuticals Sverige AB Tlf 46-08 544 982 50 Nederland Actelion Pharmaceuticals Nederland B.V. Tel 31-0348 435950

Deutschland Actelion Pharmaceuticals Deutschland GmbH Tel 49-0761 45 64 0 Norge Actelion Pharmaceuticals Sverige AB Tlf 46-08 544 982 50

Eesti Nycomed SEFA AS Tel 372 6112 569 Österreich Actelion Pharmaceuticals Austria GmbH Tel 43-01 505 4527

Actelion Pharmaceuticals E .. 30 210 675 25 00 Polska Actelion Pharma Polska Sp. z o.o. Tel 48 0 500 145 920

España Actelion Pharmaceuticals España S.L. Tel 34 93 253 10 64 Portugal Actelion Pharmaceuticals Portugal Lda. Tel 351 21 358 6120

France Actelion Pharmaceuticals France SAS Tél 33-01 58 62 32 32 România Geneva Romfarm Internacional Tel 40 021 231 3561

Ireland Actelion Pharmaceuticals UK Ltd Tel 353 1890 771 648 Slovenija Medis d.o.o. Tel 386-01 589 69 00

Ísland Actelion Pharmaceuticals Sverige AB Sími 46-08 544 982 50 Slovenská republika Actelion Pharmaceuticals SK, s.r.o. Tel 420 2 21 968 006

Italia Actelion Pharmaceuticals Italia S.r.l. Tel 39 0542 64 87 40 SuomiFinland Actelion Pharmaceuticals Sverige AB PuhTel 46-08 544 982 50

Actelion Pharmaceuticals E .. 30 210 675 25 00 Sverige Actelion Pharmaceuticals Sverige AB Tel 46-08 544 982 50

Latvija Nycomed Latvija Tel 371 784 0082 United Kingdom Actelion Pharmaceuticals UK Ltd Tel 44-0845 075 0555

Lietuva
Nycomed atstovyb-
Tel: +370 5210 9070

This leaflet was last approved in:

This medicine has been authorised under ?Exceptional Circumstances?. This means that because of the rarity of this disease it has been impossible to get complete information on this medicine. The European Medicines Agency will review any new information on the medicine every year and this leaflet will be updated as necessary.