MIGLUSTAT GEN.ORPH 100 mg HARD CAPSULES

Introduction

Package Leaflet: Information for the User

Miglustat Gen.Orph 100 mg hard capsules EFG

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 package leaflet:

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

1. What is Miglustat Gen.Orph and what is it used for

Miglustat Gen.Orph 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:

• Miglustat Gen.Orph 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). Miglustat Gen.Orph will only be used in cases where enzyme replacement therapy is not suitable for the patient.

• Miglustat Gen.Orph 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 glucosylceramide 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.

Miglustat Gen.Orph 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 Miglustat Gen.Orph

Do not take Miglustat Gen.Orph

• 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 Miglustat Gen.Orph

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

Your doctor will perform the following tests before and during your treatment with Miglustat Gen.Orph:

• 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 a reduction in body weight during treatment with Miglustat Gen.Orph. The tests will help your doctor determine if these effects are due to your disease or to pre-existing conditions or are side effects of Miglustat Gen.Orph (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 Miglustat Gen.Orph 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 Miglustat Gen.Orph 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 Miglustat Gen.Orph 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 Miglustat Gen.Orph. They may decrease the amount of Miglustat Gen.Orph in your body.

Pregnancy, breastfeeding, and fertility

Do not take Miglustat Gen.Orph 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 Miglustat Gen.Orph. Do not breastfeed while taking Miglustat Gen.Orph.

Male patients must use a reliable contraceptive method during treatment with Miglustat Gen.Orph and for three months after stopping treatment.

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

Driving and using machines

Miglustat Gen.Orph may cause dizziness. Do not drive or use tools or machines if you feel dizzy.

3. How to take Miglustat Gen.Orph

Always take this medicine exactly as your doctor has told you.

If you are not sure, talk to your doctor or pharmacist.

• For type 1 Gaucher disease:In adults, the usual 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 of age), the usual dose is two 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 of age, 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 may reduce the dose of Miglustat Gen.Orph to one capsule (100 mg) one or two times a day if you experience diarrhea during treatment with Miglustat Gen.Orph (see section 4). Your doctor will tell you how long to take the treatment.

Miglustat Gen.Orph can be taken with or without food. Swallow the capsule whole with a glass of water.

If you take more Miglustat Gen.Orph than you should

If you take more capsules than you should, talk to your doctor immediately. In clinical trials, miglustat has been used at doses ten times higher than the recommended dose: this caused a reduction in blood leukocytes and other side effects similar to those described in section 4.

If you forget to take Miglustat Gen.Orph

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

If you stop taking Miglustat Gen.Orph

Do not stop taking Miglustat Gen.Orph 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 is a common side effect).These could be signs of peripheral neuropathy, due to side effects of Miglustat Gen.Orph or could be due to pre-existing conditions. Your doctor will perform tests before and during treatment with Miglustat Gen.Orph to assess your case (see section 2).

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

If you experiencea mild tremor, usually tremor in the hands, tellyour 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 Miglustat Gen.Orph to control the tremor.

Very common– may affect more than 1 in 10 people

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

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

Common– may affect up to 1 in 10 people

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

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

Most patients notice one or more side effects of this type, 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. He or she may reduce the dose of Miglustat Gen.Orph or prescribe other medicines to control the side effects.

Reporting of side effects

If you experience any side effects, talk to your doctor or pharmacist, even if it is not listed in this leaflet. You can also report side effects directly through 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. Storing Miglustat Gen.Orph

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 carton after “EXP”. The expiry date refers to the last day of the month shown.

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. These measures will help protect the environment.

6. Contents of the pack and other information

Composition of Miglustat Gen.Orph

The active substanceis miglustat 100 mg.

The other ingredients aresodium starch glycolate (type A),

povidone (K30)

magnesium stearate

gelatin,

titanium dioxide

Appearance of the product and pack size

Miglustat Gen.Orph 100 mg hard capsules are hard gelatin capsules, white in color, with the cap and body opaque, size 4, and approximately 14.0 mm in length.

Box of 12 blisters, each blister containing 7 capsules, for a total of 84 capsules.

Marketing authorization holder:

Gen.Orph

185 Bureaux de la Colline

92213 Saint Cloud Cedex

France

Manufacturer:

Delpharm Reims

10 rue Colonel Charbonneaux

51100 Reims

France

You can obtain further information on this medicine by contacting the local representative of the marketing authorization holder.

Date of last revision of this leaflet: {month YYYY}.

Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu. There are also links to other websites on rare diseases and orphan medicines.