Miglustat DIPharma 100 mg hard capsules EFG

Package leaflet: Information for the user

Introduction

Package leaflet: information for the user

Miglustat Dipharma 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 must not be given to other people, even if they have the same symptoms as you, because it may harm them.
• 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.

What is in this leaflet

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

1. What Miglustat Dipharma is and what it is used for

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

• Miglustat Dipharma 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 bone disease.

The standard treatment for type 1 Gaucher disease is enzyme replacement therapy (ERT). Miglustat Dipharma will only be used in cases where enzyme replacement therapy is considered unsuitable for the patient.

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

If you have Niemann-Pick type C disease, lipids such as glycosphingolipids can accumulate in the cells of your brain. This may lead to neurological impairment, including slowed eye movements, problems with balance, swallowing, memory, or seizures.

Miglustat Dipharma 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 taking Miglustat Dipharma

Do not take Miglustat Dipharma:

• 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 Dipharma

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

Your doctor will carry out the following tests before and during treatment with Miglustat Dipharma:

• 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 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 Dipharma. 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 Dipharma (see section 4 for more details).

If you have 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 Dipharma 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 experience 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 Dipharma 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, as it is not known whether it is effective in this condition.

Other medicines and Miglustat Dipharma

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

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

Pregnancy, breast-feeding and fertility

Do not take Miglustat Dipharma 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 Dipharma. You must not breast-feed while taking Miglustat Dipharma.

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

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

Driving and use of machines

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

3. How to take Miglustat Dipharma

Follow exactly the instructions given by your doctor for taking this medicine. If you are unsure, 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), for 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 2 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 Dipharma to one capsule (100 mg) once or twice a day if diarrhea occurs during treatment with Miglustat Dipharma (see section 4). Your doctor will advise you on the duration of treatment.

Miglustat Dipharma can be taken with or without food. The capsule should be swallowed whole with a glass of water.

If you take more Miglustat Dipharma than you should

If you take more capsules than prescribed, consult your doctor immediately. In clinical trials, miglustat has been used at doses up to 3000 mg, which caused a reduction in blood leukocytes and other adverse effects similar to those described in section 4.

If you forget to take Miglustat Dipharma

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

If you stop taking Miglustat Dipharma

Do not stop treatment with Miglustat Dipharma 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.

Serious adverse effects

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

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

If you experience mild tremor, usually tremor in the hands, inform your doctor as soon as possible. Tremor frequently resolves without the need to interrupt treatment. Occasionally, your doctor may need to reduce the dose or even discontinue treatment with Miglustat Dipharma to control the tremor.

Very common – may affect more than 1 in 10 people

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

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

Common – may affect up to 1 in 10 people

Common adverse effects include headache, dizziness, paraesthesia (tingling or numbness), coordination disorders, hypoesthesia (reduced sensation to touch), dyspepsia (indigestion), 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 adverse effects include muscle spasms or weakness, fatigue, chills and general 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 quite quickly. If any of these adverse effects cause you problems, consult your doctor. He or she may reduce the dose of Miglustat Dipharma or prescribe other medicines to manage the adverse effects.

Reporting of adverse effects

If you experience any adverse effects, consult 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 included in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Miglustat Dipharma

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date stated on the blister and packaging after “EXP/CAD”. 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

• The active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
• The other components are magnesium stearate, gelatin, titanium dioxide (E171), printing ink (consisting of black iron oxide (E172), propylene glycol (E1520), potassium hydroxide, shellac gum).

Appearance of the product and contents of the pack

Miglustat Dipharma is an opaque white 100 mg capsule printed in black ink with “DPH02” on the cap and “100” on the body. The capsules are available in PCTFE/PVC and aluminium blister packs, in pack sizes of 84 capsules in non-perforated blisters and 84x1 capsules in single-dose perforated blisters.

Only some pack sizes may be marketed.

Marketing Authorisation Holder

Dipharma Arzneimittel GmbH
Offheimer Weg 33
65549 Limburg a. d. Lahn
Germany

Manufacturer

Doppel Farmaceutici S.r.l
Via Volturno 48
20089 Quinto di Paderno - Rozzano (MI)
Italy

Date of the most recent review of this leaflet: {MM/YYYY}.

Other sources of information

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