Prospect: information for the user

Miglustat Gen.Orph 100 mg hard capsules EFG

miglustat

Read this prospect carefully before starting to take this medicine, as it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you have any questions, consult your doctor or pharmacist.
• This medicine has been prescribed only for you and should not be given to others even if they have the same symptoms as you, as it may harm them.
• If you experience any adverse effects, consult your doctor or pharmacist, even if they are not listed in this prospect. See section 4.

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

• Miglustat Gen.Orph 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 lead to an enlarged liver and spleen, changes in the blood, and bone problems.

The usual treatment for Gaucher's disease type 1 is enzyme replacement therapy (ERT). Miglustat Gen.Orph will only be used in cases where enzyme replacement therapy is not considered suitable for the patient.

• Miglustat Gen.Orph is also used to treat the progressive neurological symptoms of Niemann-Pick C disease in adults and children.

If you have Niemann-Pick C disease, lipids such as glucosylceramide 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.

Miglustat Gen.Orph acts by inhibiting the enzyme called glucosylceramide synthase, responsible for the first step in the synthesis of most glucosylsphingolipids.

Do not take Miglustat Gen.Orph

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

Warnings and precautions

Consult your doctor or pharmacist before starting to take 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 C disease
• 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 Miglustat Gen.Orph. The tests will help your doctor determine if these effects are due to your disease or 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 (sugar cane), or not take Miglustat Gen.Orph with food, or temporarily reduce the dose. In some cases, your doctor may prescribe medication 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 Miglustat Gen.Orph 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 type 1 Gaucher disease because its effectiveness in this disease is unknown.

Use of Miglustat Gen.Orph 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 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 intend to become pregnant. For more information, consult your doctor or pharmacist. You will need to use an effective contraceptive method while taking Miglustat Gen.Orph. Do not continue breastfeeding while taking Miglustat Gen.Orph.

Male patients should use a reliable contraceptive method during treatment with Miglustat Gen.Orph, and for three months after completing treatment.

If you are pregnant or breastfeeding, or think you may be pregnant, or intend to become pregnant, consult your doctor or pharmacist before using this medication.

Driving and operating machinery

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

Follow exactly the administration instructions for this medication as indicated by your doctor.

In case of doubt, consult your doctor or pharmacist again.

• 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 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 , your doctor will adjust the dose for Niemann-Pick C disease.

In the case of kidney problems, your doctor may indicate a lower initial dose. Your doctor may reduce the dose of Miglustat Gen.Orph to one capsule (100 mg) one or two times a day in the case of diarrhea during treatment with Miglustat Gen.Orph (see section 4). Your doctor will indicate the duration of treatment.

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

If you take more Miglustat Gen.Orph than you should

If you take more capsules than indicated, consult your doctor immediately. In clinical trials, miglustat was used at doses ten times higher than the recommended dose: this caused a reduction in white blood cells and other adverse effects similar to those described in section 4.

If you forget to take Miglustat Gen.Orph

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

If you interrupt treatment with Miglustat Gen.Orph

Do not interrupt treatment with Miglustat Gen.Orph 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 (this is often observed).These could be signs of peripheral neuropathy, due to side effects of Miglustat Gen.Orph or due to existing diseases. Your doctor will perform tests before and during treatment with Miglustat Gen.Orph to evaluate your case (see section 2).

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

If youexperienceamild tremor, usuallyhand tremor,informyour doctor as soon as possible. Tremor often resolves without the need to interrupt treatment. In some cases, your doctor may need to reduce the dose and even interrupt treatment with Miglustat Gen.Orph to control the tremor.

Very common–may affect more than 1 in 10 patients

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

If you lose some weightwhen starting treatment with Miglustat Gen.Orph, do not worry, as people generally stop losing weight with continued treatment.

Common–may affect up to 1 in 10 patients

The common side effects are headache, dizziness, paresthesias (tingling or numbness), coordination disturbances,hypoesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling dizzy), constipation, and vomiting, abdominal swelling or discomfort, and thrombocytopenia (decreased platelet levels). Neurological symptoms and thrombocytopenia may be due to underlying disease.

Other possible side effectsare muscle spasms or weakness, fatigue, chills, and a feeling 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 Miglustat Gen.Orph 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 theAppendix 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 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 unused medications. By doing so, you will help protect the environment.

Composition of Miglustat Gen.Orph

The active ingredientis miglustat 100 mg.

The other components aresodium glycolate (type A),

povidone (K30)

magnesium stearate

gelatin,

titanium dioxide

Appearance of the product and contents of the package

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

Box with 12 blisters, each blister containing 7 capsules, totaling 84 capsules.

Holder of the marketing authorization:

Gen.Orph

185 Bureaux de la Colline

92213 Saint Cloud Cedex

France

Manufacturer:

Delpharm Reims

10 rue Colonel Charbonneaux

51100 Reims

France

For more information about this medication, please contact the local representative of the marketing authorization holder.

Last review date of this leaflet: {month} {year}.

For more detailed information about this medication, please visit the website of the European Medicines Agency:http://www.ema.europa.eu.There are also links to other websites about rare diseases and orphan drugs.