Miglustat Gen.Orph 100 mg hard capsules EFG

Package leaflet: Information for the user

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 questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you only and you should not give it to other people, even if they have the same symptoms as you, because it may harm them.
• If you experience any adverse reactions, consult your doctor or pharmacist, even if they are adverse reactions not listed in this leaflet. See section 4.

Leaflet contents:

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

1. What Miglustat Gen.Orph is and what it is 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 eliminate a substance called glucosylceramide. As a result, this substance accumulates in certain cells of your immune system. This may cause your liver and spleen to become enlarged, changes in blood parameters, and problems affecting the bones.

The standard 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 considered unsuitable for the patient.

• Miglustat Gen.Orph is also used to treat 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 may accumulate in the cells of your brain. This can lead to neurological impairment, including slowed eye movements, problems with balance, swallowing, memory, or seizures.

Miglustat Gen.Orph 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 starting to take Miglustat Gen.Orph

Do not take Miglustat Gen.Orph

• 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 starting to take Miglustat Gen.Orph

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

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

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

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 whether these effects are due to your underlying disease or pre-existing conditions, or are adverse effects of Miglustat Gen.Orph (see section 4 for more details).

If you experience diarrhoea, your doctor may advise you to modify your diet to reduce intake of lactose and carbohydrates such as sucrose (cane sugar), or to avoid taking Miglustat Gen.Orph with food, or to temporarily reduce the dose. In some cases, your doctor may prescribe medications to treat diarrhoea, such as loperamide. Consult your doctor if diarrhoea does not respond to these measures or if you develop any other abdominal discomfort. In such cases, your doctor may decide to perform additional investigations.

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 administer this medicine to children and adolescents (under 18 years of age) with type 1 Gaucher disease, as it is unknown whether it is effective in this condition.

Taking Miglustat Gen.Orph with other medicines

Inform your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines.

Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Miglustat Gen.Orph. These may reduce the amount of Miglustat Gen.Orph in your body.

Pregnancy, breast-feeding and fertility

Do not take Miglustat Gen.Orph if you are pregnant or planning to become pregnant. For further information, consult your doctor or pharmacist. You must use an effective method of contraception while taking Miglustat Gen.Orph. You must not breast-feed while taking Miglustat Gen.Orph.

Male patients should use a reliable method of contraception during treatment with Miglustat Gen.Orph and for three months after stopping treatment.

If you are pregnant or breast-feeding, think you might be pregnant, or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.

Driving and using machines

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

3. How to take Miglustat Gen.Orph

Follow exactly the instructions for administering this medicine as given by your doctor.

If in doubt, consult your doctor or pharmacist again.

• For type 1 Gaucher disease: In adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon, night), representing 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 night). This corresponds to 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.

If you have kidney problems, your doctor may prescribe you a lower starting dose. Your doctor may reduce the dose of Miglustat Gen.Orph to one capsule (100 mg) once or twice a day if diarrhea occurs during treatment with Miglustat Gen.Orph (see section 4). Your doctor will advise you on the duration of treatment.

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

If you take more Miglustat Gen.Orph than you should

If you take more capsules than prescribed, consult 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 adverse effects similar to those described in section 4.

If you forget to take Miglustat Gen.Orph

Take the next capsule at the scheduled time. Do not take a double dose to make up for forgotten doses.

If you stop taking Miglustat Gen.Orph

Do not stop treatment with Miglustat Gen.Orph without consulting your doctor.

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

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everyone experiences them.

More serious adverse effects:

Some patients have experienced tingling or numbness in the hands and feet (commonly observed). These could be signs of peripheral neuropathy due to adverse effects of Miglustat Gen.Orph, or they could be due to pre-existing diseases. Your doctor will perform tests before and during treatment with Miglustat Gen.Orph to assess your condition (see section 2).

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

If you experience a slight tremor, usually tremor in the hands, inform your doctor as soon as possible. Often, the tremor resolves without the need to interrupt treatment. Occasionally, your doctor may need to reduce the dose or even interrupt treatment with Miglustat Gen.Orph to control the tremor.

Very common – may affect more than 1 in 10 patients

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

If you lose some weight when starting treatment with Miglustat Gen.Orph, do not be concerned; usually people stop losing weight as treatment continues.

Common – may affect up to 1 in 10 patients

Common adverse effects are headache, dizziness, paresthesia (tingling or numbness), coordination disturbances, hypoesthesia (reduced sensation to touch), dyspepsia (indigestion), nausea (feeling sick), constipation, 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 adverse effects include muscle spasms or weakness, fatigue, chills, and malaise, difficulty sleeping, forgetfulness, and decreased libido.

Most patients notice one or more of these types of adverse effects, usually when starting treatment or at various times during treatment. Most are mild and resolve fairly quickly. If any of these adverse effects cause you problems, consult your doctor. He or she may reduce the dose of Miglustat Gen.Orph or prescribe other medications to manage the adverse effects.

Reporting of adverse effects

If you experience adverse effects, talk to your doctor or pharmacist, even if they are adverse effects not listed in this leaflet. You can also report them directly through the national reporting system listed in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Miglustat Gen.Orph

Keep this medicine out of sight and reach of children.

Do not use this medicine after the expiry date stated on the packaging after "EXP". The expiry date refers to the last day of the month indicated.

This medicine does not require any special storage conditions.

Medicines must not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and packaging that you no longer use. This will help protect the environment.

6. Contents of the pack and other information

Composition of Miglustat Gen.Orph

The active substance is miglustat 100 mg.

The other components are sodium starch glycolate (type A),
povidone (K30),
magnesium stearate,
gelatin,
titanium dioxide.

Appearance of the product and contents of the pack

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

Pack: 12 blisters, each blister containing 7 capsules, making 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

For further information about this medicinal product, please contact the local representative of the Marketing Authorization Holder.

Date of the most recent review 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.