Miglustat Dipharma

Package leaflet: Information for the user

Miglustat Dipharma 100 mg hard capsules

miglustat
Please read this leaflet carefully before 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. Do not give it to other people, even if they have the same symptoms as you, because it could be harmful.
• If you experience any side effects, including those not listed in this leaflet, contact your doctor or pharmacist. See section 4.

Contents of 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 diseases:

• Miglustat Dipharma is used to treat mild to moderate type 1 Gaucher disease in adults. Type 1 Gaucher disease is characterized by the accumulation in certain immune system cells of a substance called glucosylceramide that is not eliminated from your body. This accumulation may lead to enlargement of the liver and spleen, abnormalities in blood cells, and bone disease. The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Miglustat Dipharma is used only if a patient is considered unsuitable for treatment with enzyme replacement therapy.
• Miglustat Dipharma is also used to treat progressive neurological symptoms in Niemann-Pick disease type C in adults and children.

If you have Niemann-Pick disease type C, fats (such as glycosphingolipids) build up inside brain cells. This can cause neurological problems such as: slowed eye movements, impaired balance, swallowing difficulties, memory problems, and 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 taking Miglustat Dipharma:

• if you have kidney disease;
• if you have liver disease.

Before and during treatment with Miglustat Dipharma, your doctor will carry out the following checks:

• an examination to assess the condition of the nerves in your arms and legs;
• measurement of vitamin B levels;
• monitoring of growth in case you are a pediatric or adolescent patient with Niemann-Pick disease type C;
• monitoring of blood platelet count.

The reason these tests are performed is that, during treatment with Miglustat Dipharma,
some patients have experienced tingling or numbness in their hands and feet, or a decrease in body weight. These
tests will help your doctor determine whether such effects are due to your underlying disease or other pre-existing
conditions, or to side effects of Miglustat Dipharma (for further information see section 4).
If you have diarrhoea, your doctor may: decide to modify your diet to reduce lactose and carbohydrate intake such as sucrose (cane sugar); decide not to take Miglustat Dipharma together with food; decide to temporarily reduce the dose of Miglustat Dipharma. In some cases, your doctor may prescribe antidiarrheal medicines such as loperamide. If your diarrhoea does not respond to the above measures or if you have any other abdominal symptoms, consult your doctor. In such cases, your doctor may decide to perform further investigations.
Male patients must use reliable contraceptive methods during treatment with Miglustat Dipharma and for 3 months after completion of 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 not known whether the medicine is effective in this condition.
Other medicines and Miglustat Dipharma
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Inform your doctor if you are taking other medicines containing imiglucerase, which are sometimes used concurrently with Miglustat Dipharma. These may reduce your body's exposure to Miglustat Dipharma.
Pregnancy, breastfeeding and fertility
If you are pregnant, think you may be pregnant, or are planning a pregnancy, you must not take Miglustat Dipharma. Your doctor can provide you with further information. While taking Miglustat Dipharma, you must use an effective method of contraception. Do not breastfeed while taking Miglustat Dipharma.
Male patients must continue to use reliable contraceptive methods during treatment with Miglustat Dipharma and for 3 months after stopping treatment.
If you are pregnant, think you may be pregnant, are planning a pregnancy, or are breastfeeding, ask your doctor or pharmacist for advice before taking this medicine.
Driving and using machines
Miglustat Dipharma may cause dizziness. If you feel dizzy, do not drive or operate tools or machinery.

3. How to take Miglustat Dipharma

Take this medicine exactly as instructed by your doctor. If you have any doubts, consult your doctor or pharmacist.

• For type 1 Gaucher disease: The usual dose for adults is one capsule (100 mg) three times a day (morning, afternoon, and evening), up to a maximum of three capsules per day (300 mg).
• For Niemann-Pick disease type C: For adults and adolescents (over 12 years of age), the usual dose is 2 capsules (200 mg) three times a day (morning, afternoon, and evening), corresponding to a maximum daily dose of six capsules (600 mg). For children under 12 years of age, the doctor will appropriately adjust the dose required for Niemann-Pick disease type C. If you have kidney problems, you may initially receive a lower dose. Your doctor may decide to reduce the dose; for example, one capsule (100 mg) once or twice a day if you experience diarrhoea while taking Miglustat Dipharma (see section 4). Your doctor will decide and inform you about how long the treatment should last.

Miglustat Dipharma can be taken either on an empty stomach or with food. The capsule must 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. Miglustat has been used in clinical studies at doses up to 3,000 mg, which caused reductions in white blood cell counts and other adverse effects similar to those described in section 4.
If you forget to take Miglustat Dipharma
Take the next capsule at your usual time. Do not take a double dose to make up for a missed dose.
If you stop taking Miglustat Dipharma
Do not stop taking Miglustat Dipharma without first talking to your doctor.
If you have any questions about the use of this medicine, consult your doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine may cause side effects, although not everybody gets them.

Serious side effects
Some patients have experienced tingling or numbness in the hands and feet (commonly observed).
These could be signs of peripheral neuropathy, caused by side effects of Miglustat Dipharma or possibly due to pre-existing conditions. To evaluate these symptoms, your doctor will perform certain tests before and during treatment with Miglustat Dipharma (see section 2).
If you experience any of these symptoms, please seek medical advice from your doctor as soon as possible.
If you experience a slight tremor, usually trembling of the hands, please seek medical advice from your doctor as soon as possible. The tremor often disappears without needing to stop treatment.
Sometimes, your doctor may need to reduce the dose or discontinue treatment with Miglustat Dipharma to make the tremor go away.

Very common side effects – may affect more than 1 in 10 people
The most commonly reported side effects include: diarrhoea, flatulence (excess intestinal gas), abdominal pain (stomach), weight loss, and reduced appetite.
If you lose weight when starting treatment with Miglustat Dipharma, do not be concerned. Weight loss usually stops as treatment continues.

Common side effects – may affect up to 1 in 10 people
Common side effects of treatment include headache, dizziness, paresthesia (tingling or numbness), abnormal coordination, hypoesthesia (reduced sensitivity to touch), dyspepsia (indigestion), nausea (feeling unwell), constipation and vomiting, abdominal bloating or discomfort (gastric), thrombocytopenia (reduced levels of platelets in the blood).

Neurological symptoms and thrombocytopenia may be due to the underlying disease.

Other possible side effects include: muscle weakness or spasms, fatigue, chills and malaise, depression, sleep difficulties, memory loss, and decreased libido.

Most patients experience one or more of these side effects, usually at the beginning of treatment or intermittently during treatment. These cases are mostly mild and disappear quickly. If any of these side effects cause discomfort, consult your doctor. The doctor may reduce your dose of Miglustat Dipharma or recommend other medicines to help counteract the side effects.

Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, talk to your doctor or pharmacist. You can also report side effects directly via the national reporting system detailed in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store 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 on the carton after the word “EXP”. The expiry date refers to the last day of that month.
This medicine does not require any special storage conditions.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

6. Package contents and other information

What Miglustat Dipharma contains

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

Description of the appearance of Miglustat Dipharma and package contents
Miglustat Dipharma is a 100 mg opaque white hard capsule with "DPH02" printed in black on the cap and "100" printed in black on the body.
The capsules are presented in PCTFE/PVC/aluminum blisters, in packs of 84 hard capsules in non-perforated blisters in cardboard box or in wallet packaging, and 84x1 hard capsules in unit-dose divisible blisters in cardboard box.
Not all pack sizes may be marketed.

Marketing Authorization Holder and Manufacturer
Marketing Authorization Holder:
Dipharma Arzneimittel GmbH
Offheimer Weg 33
65549 Limburg a. d. Lahn
Germany

Manufacturer:
Doppel Farmaceutici S.r.l.
Via Volturno 48
20089 Quinto de' Stampi – Rozzano (MI)
Italy

Other sources of information
More detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu.