Label: information for the user

Zavesca 100 mg capsules

miglustat

Read this label carefully before starting to take this medicine, as it contains important information for you.

• Keep this label, as you may need to read it again.
• If you have any questions, consult your doctor or pharmacist.
• This medicine has been prescribed only for you and should not be given to others even if they have the same symptoms as you, as it may harm them.
• If you experience any side effects, consult your doctor or pharmacist, even if they are not listed in this label. See section 4.

Zavesca contains the active substance miglustat, which belongs to a group of medicines that affect metabolism. It is used to treat two disorders:

• Zavesca is used to treat mild-moderate Gaucher's disease type 1 in adults.

In Gaucher's disease type 1, your body cannot eliminate a substance called glucosylceramide. As a result, it accumulates in some cells of your immune system. This can lead to an enlarged liver and spleen, changes in the blood, and bone problems.

The usual treatment for Gaucher's disease type 1 is enzyme replacement therapy (ERT). Zavesca will only be used in cases where enzyme replacement therapy is not considered suitable for the patient.

• Zavesca is also used to treat the progressive neurological symptoms of Niemann-Pick disease type C in adults and children.

If you have Niemann-Pick disease type C, lipids such as glycosphingolipids may accumulate in the cells of your brain. This can lead to neurological function alterations such as slow eye movements, balance, swallowing, memory, or seizures.

Zavesca acts by inhibiting the enzyme called glucosylceramide synthase, responsible for the first step in the synthesis of most glycosphingolipids.

Do not take Zavesca

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

Warnings and precautions

Consult your doctor or pharmacist before starting to take Zavesca

• if you have kidney disease
• if you have liver disease

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

• physical examination of the nerves in your arms and legs
• measurement of your vitamin B12 levels
• monitoring of growth in children or adolescents with Niemann-Pick type C disease
• monitoring of your platelet count in blood

These tests are necessary because some patients have reported symptoms such as tingling or numbness in hands and feet, or a reduction in body weight during treatment with Zavesca. The tests will help your doctor determine if these effects are due to your disease or existing conditions or are side effects of Zavesca (see section 4 for more details).

If you have diarrhea, your doctor may ask you to modify your diet to reduce lactose and carbohydrate intake such as sucrose (sugar cane), or not take Zavesca with food, or temporarily reduce the dose. In some cases, your doctor may prescribe medication to treat diarrhea, such as loperamide. Consult your doctor if diarrhea does not respond to these measures or if you experience any other abdominal discomfort. In this case, your doctor may decide to perform additional tests.

It is recommended that male patients use reliable contraceptive methods during treatment with Zavesca and for three months after completing treatment.

Children and adolescents

Do not administer this medicine to children and adolescents (under 18 years of age) with Gaucher disease type 1 because its effectiveness in this disease is unknown.

Other medicines and Zavesca

Inform your doctor or pharmacist if you are taking or have taken recently or may need to take any other medicine.

Inform your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Zavesca. They may decrease the amount of Zavesca in your body.

Pregnancy, breastfeeding, and fertility

Do not take Zavesca if you are pregnant or intend to become pregnant. For more information, consult your doctor or pharmacist. You will need to use an effective contraceptive method while taking Zavesca. Do not continue breastfeeding while taking Zavesca.

Male patients should use a reliable contraceptive method during treatment with Zavesca, and for three months after completing treatment.

If you are pregnant or breastfeeding, think you may be pregnant, or intend to become pregnant, consult your doctor or pharmacist before using this medicine.

Driving and operating machinery

Zavesca may cause dizziness. Do not drive or operate tools or machinery if you feel dizzy.

Zavesca contains sodium

This medicine contains less than 1 mmol of sodium (23 mg) per capsule; it is essentially "sodium-free".

Follow exactly the administration instructions for this medication as indicated by your doctor. In case of doubt, consult your doctor or pharmacist again.

• For Type 1 Gaucher's disease : In adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon, night), which represents a maximum daily dose of three capsules (300 mg).

• For Niemann-Pick type C disease: In adults and adolescents (over 12 years), the usual dose is 2 capsules (200 mg) three times a day (morning, afternoon, and night). This represents a maximum daily dose of six capsules (600 mg).

For children under 12 years, your doctor will adjust the dose for Niemann-Pick type C disease.

In the case of kidney problems, your doctor may indicate a lower initial dose. Your doctor may reduce the Zavesca dose to one capsule (100 mg) once or twice a day if diarrhea occurs during treatment with Zavesca (see section 4). Your doctor will indicate the duration of treatment.

To remove the capsule:

1. Separate along the perforated line
2. Remove the paper according to the arrows
3. Extract the capsule by pressing through the aluminum

Zavesca can be taken with or without food. Swallow the entire capsule with a glass of water.

If you take more Zavesca than you should

If you take more capsules than indicated, consult your doctor immediately. In clinical trials, Zavesca has been used at doses of up to 3000 mg: this caused a reduction 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 when due. Do not take a double dose to compensate for the missed doses.

If you interrupt treatment with Zavesca

Do not stop treatment with Zavesca without consulting your doctor.

If you have any other questions about the use of this medication, ask your doctor or pharmacist.

Like all medicines, this medicine can cause side effects, although not everyone will experience them.

More serious side effects:

Some patients have experienced tingling or numbness in their hands and feet (this is observed frequently).These could be signs of peripheral neuropathy, due to side effects of Zavesca or could be due to existing diseases. Your doctor will perform tests before and during treatment with Zavesca to evaluate your case (see section 2).

Inform your doctor as soon as possible if you experience any of these side effects.

If youexperienceamild tremor, usuallytremor in the hands, informyour doctor as soon as possible. Tremor often resolves without the need to interrupt treatment. In some cases, your doctor may need to reduce the dose and even interrupt treatment with Zavesca to control the tremor.

Very common:(can affect more than 1 in 10 patients)

Among the most frequent side effects are diarrhea, flatulence (gas), abdominal pain (stomach), weight loss, and decreased appetite.

If you lose some weightwhen starting treatment with Zavesca, do not worry, usually people stop losing weight with continued treatment.

Common:(can affect up to 1 in 10 patients)

Common side effects are headache, dizziness, paresthesias (tingling or numbness), coordination disturbances, hypoesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling sick), constipation, and vomiting, abdominal swelling or discomfort, and thrombocytopenia (decreased platelet levels). Neurological symptoms and thrombocytopenia may be due to underlying disease.

Other possible side effects are muscle spasms or weakness, fatigue, chills, and feeling unwell, depression, difficulty sleeping, forgetfulness, and decreased libido.

Most patients notice one or more of these side effects, usually when starting treatment or at different times during treatment. Most are mild and resolve quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of Zavesca or prescribe other medications to control side effects.

Reporting side effects

Ifyou experience side effects, consult your doctor or pharmacist, even if they are side effects that do not appear in this prospectus. You can also report them directly through thenational notification system included in theAnnex V. By reporting side effects, you can contribute to providing more information about the safety of this medicine.

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

Do not use this medication after the expiration date that appears on the packaging after “CAD”. The expiration date is the last day of the month indicated.

Do not store at a temperature above 30°C.

Medicines should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the packaging and medications that you no longer need. In this way, you will help protect the environment.

Composition of Zavesca

The active ingredientis miglustat 100 mg.

The other components are:

Sodium glycolate starch,

Povidone (K30),

Magnesium stearate.

Gelatin,

Titanium dioxide (E171).

Iron oxide black (E172),

Shellac.

Appearance of the product and contents of the package

Zavesca is a white capsule of 100 mg with “OGT 918” printed in black on the cap and “100” printed in black on the body.

Box with four blister strips, containing each strip 21 capsules, totaling 84 capsules.

Marketing Authorization Holder:

Janssen-Cilag International NV

Turnhoutseweg 30

B-2340 Beerse

Belgium

Responsible for manufacturing:

Janssen Pharmaceutica NV

Turnhoutseweg 30

B-2340 Beerse

Belgium

For more information about this medication, please contact the local representative of the marketing authorization holder.

Last review date of this leaflet:

The detailed information about this medication is available on the website of the European Medicines Agency:http://www.ema.europa.eu.There are also links to other websites about rare diseases and orphan drugs.