Zavesca - patient leaflet, side effects, dosage

1. What Zavesca is and what it is used for

Zavesca contains the active substance miglustat which 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 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. 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 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.

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

2. What you need to know before you take Zavesca

Do not take Zavesca

• – 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 Zavesca

• – if you suffer from kidney disease

• – if you suffer from liver disease

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 B12 levels

• – monitoring growth if you are a child or adolescent with Niemann-Pick type C disease

• – monitoring of blood platelet counts

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 such as sucrose (cane sugar), 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.

Children and adolescents

Do not give this medicine to children and adolescents (below 18 years old) with type 1 Gaucher disease because it is not known if it works in this disease.

Other medicines and Zavesca

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 medicines containing imiglucerase, which are sometimes used at the same time as Zavesca. They may lower the amount of Zavesca in your body.

Pregnancy, breast-feeding and fertility

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

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

If you are pregnant, breast feeding, think you may be pregnant or planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

Driving and using machines

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

Zavesca contains sodium

This medicine contains less than 1 mmol sodium (23 mg) per capsule, that is to say essentially ‘sodium-free’.

3. How to take Zavesca

Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.

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 (over 12 years old), 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).

For children less than 12 years old , your doctor will adjust the 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:

—(2>------ ----®---

• 1. Separate at perforations

• 2. Peel back paper at arrows

• 3. Push product through foil

4. Possible side effects

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

Most serious side effects:

Some patients have had tingling or numbness in the hands and feet (seen commonly). 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 seek medical advice from your doctor as soon as possible.

If you get a slight tremor , usually trembling hands, seek medical advice from 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.

Very common: ( may affect more than 1 in 10 people)

The most common side effects are diarrhoea, flatulence (wind), abdominal (stomach) pain, weight loss and decreased appetite.

If you do lose some weight when you start treatment with Zavesca don’t worry. People usually stop losing weight as treatment goes on.

Common: ( may affect up to 1 in 10 people)

Common side effects of treatment include headache, dizziness, 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 chills and malaise, depression, difficulty sleeping, forgetfulness, 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.

Reporting of side effects

If you get any side effects, talk to your doctor or pharmacist . This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V. By reporting side effects you can help provide more information on the safety of this medicine.

5. How to store Zavesca

Keep this medicine out of the sight and reach of children.

Do not take this medicine after the expiry date which is stated on the carton after ‘EXP’. The expiry date refers to the last day of that month.

Do not store above 30°C.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer take. These measures will help protect the environment.

6. Contents of the pack and other information

What Zavesca contains

The active substance is miglustat 100 mg.

The other ingredients are:

Sodium starch glycollate,

Povidone (K30),

Magnesium stearate.

Gelatin, Titanium dioxide (E171).

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:

Janssen-Cilag International NV

Turnhoutseweg 30

B-2340 Beerse

Belgium

Manufacturer:

Janssen Pharmaceutica NV

Turnhoutseweg 30

B-2340 Beerse

Belgium

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

This leaflet was last revised in:

Detailed information on this medicine is available on the European Medicines Agency website:. There are also links to other websites about rare diseases and treatments.

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