Zavesca

Package leaflet: Information for the user

Zavesca 100 mg capsules

miglustat
Please 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 they have the same symptoms as yours, as it may be harmful. If you experience any side effects, including those not listed in this leaflet, tell your doctor or pharmacist. See section 4.

Contents of this leaflet:

1. What Zavesca is and what it is used for.
2. What you need to know before taking Zavesca.
3. How to take Zavesca.
4. Possible side effects.
5. How to store Zavesca.
6. Contents of the pack and other information.

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 diseases:

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

Type 1 Gaucher disease is characterised by the accumulation, in certain cells of the immune system, of a substance called glucosylceramide that is not eliminated from the body. This accumulation can lead to enlargement of the liver and spleen, abnormalities in blood cells, and bone disease.
The standard treatment for type 1 Gaucher disease is enzyme replacement therapy. Zavesca is used only if a patient is considered unsuitable for treatment with enzyme replacement therapy.

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

If you have Niemann-Pick disease type C, fats (such as glycosphingolipids) accumulate within brain cells. This can cause neurological problems affecting functions such as eye movement, balance, swallowing, memory, and seizures.
Zavesca 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 Zavesca

Do not take Zavesca:

• 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 Zavesca:

• if you have kidney problems;
• if you have liver problems.

Before and during treatment with Zavesca, your doctor will carry out the following checks:

• examination to assess the condition of the nerves in your arms and legs;
• measurement of vitamin B levels;
• monitoring of growth in case you are a paediatric or adolescent patient with Niemann-Pick disease type C;
• monitoring of blood platelet count.

The reason these tests are performed is that, during treatment with Zavesca, some patients have experienced tingling or numbness in their hands and feet, or a decrease in body weight. These tests will help your doctor determine whether such effects are due to your underlying disease or other pre-existing conditions, or to side effects of Zavesca (for further information, see section 4).
If you have diarrhoea, your doctor may: decide to modify your diet to reduce lactose and intake of carbohydrates such as sucrose (cane sugar); decide not to take Zavesca with food; decide to temporarily reduce the dose of Zavesca. In some cases, your doctor may prescribe anti-diarrhoeal medicines such as loperamide. Cases of Crohn's disease (an inflammatory disease affecting the intestine) have been reported in patients with Niemann-Pick disease type C treated with Zavesca. 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 carry out further investigations to determine whether your symptoms may be due to another cause.
Male patients should use reliable contraceptive methods during treatment with Zavesca and for 3 months after stopping the treatment.

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 disease.

Other medicines and Zavesca
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 concomitantly with Zavesca. These may reduce your body's exposure to Zavesca.

Pregnancy, breast-feeding and fertility
If you are pregnant, think you might be pregnant, or are planning a pregnancy, you must not take Zavesca. Your doctor will be able to provide you with further information. You must use an effective method of contraception during treatment with Zavesca. Do not breast-feed while taking Zavesca.
Male patients must continue to use reliable contraceptive methods during treatment with Zavesca and for 3 months after stopping treatment.
If you are pregnant, think you might be pregnant, planning a pregnancy, or are breast-feeding, ask your doctor or pharmacist for advice before taking this medicine.

Driving and using machines
Zavesca may cause dizziness. If you feel dizzy, do not drive or operate machinery.

Zavesca contains sodium
This medicine contains less than 1 mmol (23 mg) of sodium per capsule, i.e. essentially 'sodium-free'.

3. How to take Zavesca

Take this medicine exactly as prescribed by your doctor. If you have any doubts, consult your doctor or pharmacist.

• For type 1 Gaucher disease: The usual dose for adults is one capsule (100 mg) three times a day (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 a day (morning, afternoon, and evening), for 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 a day if you develop diarrhoea while taking Zavesca (see section 4). Your doctor will decide and inform you how long the treatment should last.

To remove the capsule:

1. Tear along the perforated line
2. Remove the paper at the arrows
3. Press the product out from the foil

Zavesca can be taken with or without food. The capsule must be swallowed whole with a glass of water.

If you take more Zavesca than you should
If you take more capsules than prescribed, contact your doctor immediately. Zavesca has been used in clinical studies at doses up to 3000 mg, which caused reductions in white blood cell counts and other adverse effects similar to those described in section 4.

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

If you stop taking Zavesca
Do not stop taking Zavesca without first talking to your doctor.

If you have any questions about 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 everyone experiences 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 Zavesca or possibly due to underlying conditions. To assess these symptoms, your doctor will perform certain tests before and during treatment with Zavesca (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 trembling of the hands , seek medical advice from your doctor as soon as possible. The tremor often disappears without the need to stop treatment.
Sometimes your doctor may need to reduce the dose or discontinue treatment with Zavesca to make the tremor go away.
Very common: ( may affect more than 1 in 10 people)
Among the most common side effects are: diarrhoea, flatulence (excessive intestinal gas), abdominal pain (stomach), weight loss and reduced appetite.
If you lose weight when starting treatment with Zavesca, do not be concerned. Weight loss usually stops as treatment continues.
Common: ( 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 unwell), constipation and vomiting, abdominal swelling or discomfort (gastric), thrombocytopenia (reduced platelet levels in the blood).
Neurological symptoms and thrombocytopenia may be due to the underlying disease.
Other possible side effects include: muscle weakness or spasms, fatigue, chills and malaise, depression, sleep disturbances, 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 resolve quickly. If any of these side effects cause discomfort, consult your doctor. The doctor may reduce your dose of Zavesca or recommend other medicines to help counteract the side effects.
Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, talk to your doctor or pharmacist. You can also report side effects directly via 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 Zavesca

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 container after the word
"Exp.". The expiry date refers to the last day of that month.
Do not store above 30 °C.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Package contents and other information

What Zavesca contains
The active substance is miglustat 100 mg.
The other components are:
Sodium starch glycolate,
Povidone (K30),
Magnesium stearate.
Gelatin,
Titanium dioxide (E171).
Black iron oxide (E172),
Shellac.
Description of the appearance of Zavesca and contents of the pack
Zavesca is a white 100 mg capsule with the black imprint “OGT 918” on the cap and the number “100”
in black on one side.
One pack contains 4 blisters; each blister contains 21 capsules, for a total of 84 capsules per
pack.
Marketing Authorization Holder
Janssen-Cilag International NV
Turnhoutseweg 30
B-2340 Beerse
Belgium
Manufacturer:
Janssen Pharmaceutica NV
Turnhoutseweg 30
B-2340 Beerse
Belgium
For further information about this medicinal product, please contact the local representative of the Marketing Authorization Holder.
België/Belgique/Belgien Lietuva
Janssen-Cilag NV UAB “JOHNSON & JOHNSON”
Tel/Tél: +32 14 64 94 11 Tel: +370 5 278 68 88
[email protected] [email protected]
l
България Luxembourg/Luxemburg
„Джонсън & Джонсън България” ЕООД Janssen-Cilag NV
Тел.: +359 2 489 94 00 Tél/Tel: +32 14 64 94 11
[email protected] [email protected]
Česká republika Magyarország
Janssen-Cilag s.r.o. Janssen-Cilag Kft.
Tel: +420 227 012 227 Tel.: +36 1 884 2858
[email protected]
Danmark Malta
Janssen-Cilag A/S AM MANGION LTD
Tlf.: +45 4594 8282 Tel: +356 2397 6000
[email protected]
Deutschland Nederland
Janssen-Cilag GmbH Janssen-Cilag B.V.
Tel: 0800 086 9247 / +49 2137 955 6955 Tel: +31 76 711 1111
[email protected] [email protected]
Eesti Norge
UAB “JOHNSON & JOHNSON” Eesti filiaal Janssen-Cilag AS
Tel: +372 617 7410 Tlf: +47 24 12 65 00
[email protected] [email protected]
e
Ελλάδα Österreich
Janssen-Cilag Φαρμακευτική Μονοπρόσωπη Janssen-Cilag Pharma GmbH
Α.Ε.Β.Ε. Tel: +43 1 610 300
Tηλ: +30 210 80 90 000 T
T
España Polska
Janssen-Cilag, S.A. Janssen-Cilag Polska Sp. z o.o.
Tel: +34 91 722 81 00 Tel.: +48 22 237 60 00
[email protected]
France Portugal
Janssen-Cilag Janssen-Cilag Farmacêutica, Lda.
Tél: 0 800 25 50 75/+33 1 55 00 40 03 Tel: +351 214 368 600
[email protected]
Hrvatska România
Johnson & Johnson S.E. d.o.o. Johnson & Johnson România SRL
Tel: +385 1 6610 700 Tel: +40 21 207 1800
[email protected]
Ireland Slovenija
Janssen Sciences Ireland UC Johnson & Johnson d.o.o.
Tel: 1 800 709 122 Tel: +386 1 401 18 00
[email protected] [email protected]
Ísland Slovenská republika
Janssen-Cilag AB Johnson & Johnson, s.r.o.
c/o Vistor hf. Tel: +421 232 408 400
Sími: +354 535 7000
[email protected]
Italia Suomi/Finland
Janssen-Cilag SpA Janssen-Cilag Oy
Tel: 800.688.777/+39 02 2510 1 Puh/Tel: +358 207 531 300
[email protected] [email protected]
Κύπρος Sverige
Βαρνάβας Χατζηπαναγής Λτδ Janssen-Cilag AB
Τηλ: +357 22 207 700 Tfn: +46 8 626 50 00
[email protected]
Latvija United Kingdom (Northern Ireland)
UAB “JOHNSON & JOHNSON” filiāle Latvijā Janssen Sciences Ireland UC
Tel: +371 678 93561 Tel: +44 1 494 567 444
[email protected] [email protected]
More detailed information about this medicinal product is available on the website of the European Medicines Agency: https://www.ema.europa.eu. In addition, links to other websites on rare diseases and their therapeutic treatments are provided.