Leaflet: information for the user

Yargesa 100 mg hard capsules

miglustat

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

Keep this leaflet, as 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 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 leaflet. See section 4.

What Yargesa is and what it is used for.

What you need to know before you start taking Yargesa.

How to take Yargesa.

Possible side effects.

Storage of Yargesa.

Contents of the pack and additional information.

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

• Yargesa 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 cause your liver and spleen to become enlarged, changes in your blood, and affect your bones.

The usual treatment for Gaucher's disease type 1 consists of enzyme replacement therapy (ERT). Yargesa will only be used in cases where enzyme replacement therapy is not considered 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 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.

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

Do not take Yargesa

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

Warnings and precautions

Consult your doctor or pharmacist before starting to take 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 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 this medication. The tests will help your doctor determine if these effects are due to your underlying condition or existing conditions or are adverse 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 (sugar cane), or not take Yargesa with food, or temporarily reduce the dose. In some cases, your doctor may prescribe medications 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 Yargesa and for three months after completing treatment.

Children and adolescents

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

Use of Yargesa with other medications

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

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

Male patients should use a reliable contraceptive method during treatment with this medication, 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 medication.

Driving and operating machines

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

Yargesa contains sodium

This medication 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. If in doubt, consult your doctor or pharmacist again.

Dose

• For Gaucher's disease type 1: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 two 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 old, 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 dose of Yargesa to one capsule (100 mg) once or twice a day if diarrhea occurs during treatment with Yargesa (see section 4). Your doctor will indicate the duration of treatment.

To extract the capsule:

1. Separate along the perforated line

2. Remove the paper as indicated by the arrows

3. Extract the capsule by pressing through the aluminum

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

If you take more Yargesa than you should:

If you take more capsules than indicated, consult your doctor immediately. In clinical trials, miglustat was 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 Yargesa:

Take the next capsule when due. Do not take a double dose to compensate for missed doses.

If you interrupt treatment with Yargesa:

Do not stop treatment with this medication 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 the hands and feet (observed frequently). These could be signs of peripheral neuropathy, due to side effects of Yargesa or could be due to existing diseases. Your doctor will perform tests before and during treatment with Yargesa to evaluate your case (see section 2).

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

If youexperiencealight tremor, usuallytremor in the hands, informyour doctor as soon as possible. Tremor often resolves without the need to interrupt treatment. Occasionally, your doctor may need to reduce the dose and even interrupt treatment with Yargesa to control the tremor.

Muy frecuentes– 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 Yargesa, do not worry, usually people stop losing weight with continued treatment.

Frecuentes– can affect up to 1 in 10 patients

Frequent 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 in the blood). Neurological symptoms and thrombocytopenia may be due to underlying disease.

Other possible side effects are muscle spasms or weakness, fatigue, chills, and a sense of discomfort, 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. They may reduce the dose of Yargesa or prescribe other medications to control side effects.

Reporting side effects

If you 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 theAnexo V. By reporting side effects, you can contribute to providing more information on 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 blister pack and on the packaging after “CAD”. The expiration date is the last day of the month indicated.

This medication does not require special storage conditions.

Medications 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 Yargesa

• The active ingredient is miglustat. Each hard capsule contains 100 mg of miglustat.
• The other components are

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

Capsule coating: gelatin, titanium dioxide (E171),

Printing ink is enamel, iron oxide black (E172), prolipenglicol, concentrated ammonia solution

Appearance of the product and content of the container

Yargesa is a white, hard, opaque capsule formed by a body and a cap, 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 polychlorotrifluoroethylene (PCTFE) sealed blister with an aluminum foil.

Container size of 84 x 1 hard capsules.

Marketing authorization holder and responsible manufacturer

Piramal Critical Care B.V.

Rouboslaan 32 (ground floor)

2252 TR, Voorschoten

The Netherlands

Last review date of this leaflet:<{MM/AAAA}><{month AAAA}>.

Other sources of information

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