YARGESA 100 mg HARD CAPSULES

Introduction

Package Leaflet: Information for the User

Yargesa 100 mg Hard Capsules

miglustat

Read all of this leaflet carefully before you start taking this medicine because it contains important information for you.

Keep this leaflet, you may need to read it again.

If you have any further questions, ask your doctor or pharmacist.

This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.

If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack and other information

What is Yargesa and what is it used for.

What you need to know before you take Yargesa.

How to take Yargesa.

Possible side effects.

Storing Yargesa.

Contents of the pack and further information.

1. What is Yargesa and what is it used for

Yargesa contains the active substance miglustat, which belongs to a group of medicines that have an effect on metabolism. It is used to treat two disorders:

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

In type 1 Gaucher disease, your body cannot break down a substance called glucosylceramide. As a result, it builds up in some cells of your immune system. This can lead to an enlarged liver and spleen, changes in the blood, and effects on the bones.

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

• Yargesa 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 can build up in the cells of your brain. This can lead to problems with neurological functions such as slow eye movements, balance, swallowing, memory, or seizures.

Yargesa 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 Yargesa

Do not take Yargesa

• 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 you start taking Yargesa

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

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

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

These tests are necessary because some patients have reported symptoms such as tingling or numbness in hands and feet, or weight loss during treatment with this medicine. The tests will help your doctor determine if these effects are due to your disease or pre-existing conditions or are side effects of Yargesa (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 (cane sugar), or not to take Yargesa with food, or to temporarily reduce the dose. In some cases, your doctor may prescribe medications to treat diarrhea, such as loperamide. Talk to your doctor if the 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.

Male patients are advised to use reliable contraceptive methods during treatment with Yargesa and for three months after stopping treatment.

Children and adolescents

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

Using Yargesa with other medicines

Tell your doctor or pharmacist if you are taking or have recently taken or might take any other medicines.

Tell your doctor if you are taking medicines that contain imiglucerase, which are sometimes used at the same time as Yargesa. They may decrease the amount of Yargesa in your body.

Pregnancy, breastfeeding, and fertility

Do not take Yargesa if you are pregnant or planning to become pregnant. For more information, talk to your doctor or pharmacist. You must use an effective contraceptive method while taking Yargesa. You should not breastfeed while taking Yargesa.

Male patients should use a reliable contraceptive method during treatment with this medicine and for three months after stopping treatment.

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

Driving and using machines

Yargesa may cause dizziness. Do not drive or use tools or machines if you feel dizzy.

Yargesa contains sodium

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

3. How to take Yargesa

Follow exactly the administration instructions of this medicine given by your doctor. If you are unsure, talk to your doctor or pharmacist again.

Dose

• For type 1 Gaucher disease:In adults, the normal dose is one capsule (100 mg) three times a day (morning, afternoon, evening), which is a maximum daily dose of three capsules (300 mg).

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

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

In case of kidney problems, your doctor may prescribe a lower initial dose. It is possible that your doctor will reduce the dose of Yargesa to one capsule (100 mg) one or two times a day if you experience diarrhea during treatment with Yargesa (see section 4). Your doctor will tell you how long to take the treatment.

To remove the capsule:

1. Separate at the perforated line
2. Remove the paper as indicated by the arrows
3. Remove the capsule by pressing through the aluminum

Yargesa can be taken with or without food. You should swallow the capsule whole with a glass of water.

If you take more Yargesa than you should

If you take more capsules than you were told to, talk to your doctor immediately. In clinical trials, miglustat has been used at doses of up to 3000 mg: this caused a reduction in blood leukocytes and other side effects similar to those described in section 4.

If you forget to take Yargesa

Take the next capsule when it is due. Do not take a double dose to make up for forgotten doses.

If you stop taking Yargesa

Do not stop taking this medicine without talking to your doctor.

If you have any further questions on 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 everybody gets them.

More serious side effects

Some patients have reported tingling or numbness in hands and feet (this happens frequently). These could be signs of peripheral neuropathy, due to side effects of Yargesa or could be due to pre-existing conditions. Your doctor will perform tests before and during treatment with Yargesa to assess your case (see section 2).

If you experience any of these effects, tell your doctor as soon as possible.

If you experience a mild tremor, usually a tremor in the hands, tell your doctor as soon as possible.The tremor usually resolves without needing to stop treatment. In some cases, your doctor may need to reduce the dose and even stop treatment with Yargesa to control the tremor.

Very common– may affect more than 1 in 10 people

The most common side effects include diarrhea, flatulence (gas), abdominal pain (stomach pain), weight loss, and decreased appetite.

If you lose some weightwhen you start taking Yargesa, do not worry, people usually stop losing weight with continued treatment.

Common– may affect up to 1 in 10 people

Common side effects include headache, dizziness, paresthesia (tingling or numbness), coordination problems, hypoesthesia (reduced sensation to touch), dyspepsia (indigestion), nausea (feeling sick), constipation, and vomiting, abdominal swelling or discomfort (stomach) and thrombocytopenia (low platelet count). Neurological symptoms and thrombocytopenia may be due to the underlying disease.

Other possible side effects include muscle spasms or weakness, fatigue, chills, and feeling unwell, difficulty sleeping, memory problems, and decreased libido.

Most patients experience 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 bother you, talk to your doctor. They may reduce the dose of Yargesa or prescribe other medicines to control the side effects.

Reporting side effects

If you experience side effects, talk to your doctor or pharmacist, even if they are not listed in this leaflet. 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. Storing Yargesa

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 blister and on the carton after "EXP". The expiry date is the last day of the month stated.

This medicine does not require any special storage conditions.

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

6. Contents of the pack and further information

Composition of Yargesa

• the active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
• the other ingredients are

capsule content: sodium starch glycolate (type A), povidone (K29/32), magnesium stearate,

capsule shell: gelatin, titanium dioxide (E171),

printing ink, black iron oxide (E172), propylene glycol, concentrated ammonia solution

Appearance and packaging

Yargesa is a white hard capsule with a body and cap, opaque, white, with "708" printed in black on the body. The capsule size is 4 (14.3 mm x 5.3 mm). The capsules are presented in a PVC and PCTFE blister sealed with an aluminum foil.

Pack size of 84 x 1 hard capsules.

Marketing authorization holder and manufacturer

Piramal Critical Care B.V.

Rouboslaan 32 (ground floor)

2252 TR, Voorschoten

The Netherlands

Date of last revision of this leaflet:<{MM/AAAA}><{month YYYY}>.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu.