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Miglustat Dipharma - patient leaflet, side effects, dosage

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Patient leaflet - Miglustat Dipharma

Miglustat Dipharma 100 mg hard capsules

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.

What is in this leaflet

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

  • 2. What you need to know before you take 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 conditions:

  • Miglustat Dipharma is used to treat mild to moderate type 1 Gaucher disease in adults.

In type 1 Gaucher disease, a substance called glucosylceramide is not removed from your body. It starts to build up in certain cells of the body’s immune system. This can result in liver and spleen enlargement, changes in the blood, and bone disease.

The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Miglustat Dipharma is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy.

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

If you have Niemann-Pick type C disease, fats such as glycosphingolipids build up in the cells of your brain. This can result in disturbances in neurological functions such as slow eye movements, balance, swallowing, and memory, and in seizures.

Miglustat Dipharma 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 Dipharma

Do not take Miglustat Dipharma:

  • – 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 taking Miglustat Dipharma

  • if you suffer from kidney disease
  • if you suffer from liver disease

Since some patients have had tingling or numbness in the hands and feet, or a decrease in body weight, while taking Miglustat Dipharma, your doctor will perform the following tests before treatment and during treatment with Miglustat Dipharma:

  • an examination to check the nerves in your arms and legs
  • measurement of vitamin B12 levels
  • monitoring growth if you are a child or adolescent with Niemann-Pick type C disease
  • monitoring of blood platelet counts

The tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Miglustat Dipharma (see section 4 for further details).

If you have diarrhoea, your doctor may ask you to change your diet to reduce your lactose and carbohydrate intake such as sucrose (cane sugar), or not to take Miglustat Dipharma together with food, or to temporarily reduce your dose. In some cases the doctor may prescribe anti-diarrhoeal medicines such as loperamide. If your diarrhoea does not respond to these measures, or if you have any other abdominal complaint, consult your doctor. In such case, your doctor may decide to conduct further investigations.

Male patients should use reliable birth control methods during their treatment with Miglustat Dipharma and for 3 months after finishing treatment.

Children and adolescents

Do not give this medicine to children and adolescents (below 18 years old) with type 1 Gaucher disease because it is not known if it works in this disease.

Other medicines and Miglustat Dipharma

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

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

Pregnancy, breast-feeding and fertility

You should not take Miglustat Dipharma if you are pregnant or thinking of becoming pregnant. Your doctor can give you more information. You must use effective birth control while taking Miglustat Dipharma. Do not breast-feed while you are taking Miglustat Dipharma.

Male patients should use reliable birth control methods during their treatment with Miglustat Dipharma and for 3 months after finishing treatment.

If you are pregnant, breast feeding, think you may be pregnant or planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

Driving and using machines

Miglustat Dipharma may make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy.

3. How to take Miglustat Dipharma

Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.

  • For typer 1 Gaucher disease: For adults the usual dose is one capsule (100 mg) three times a day (morning, afternoon and evening). This means a daily maximum of three capsules (300 mg).
  • For Niemann-Pick type C disease: For adults and adolescents (over 12 years old), the usual dose is two capsules (200 mg) three times a day (morning, afternoon and evening). This means a daily maximum of six capsules (600 mg).

For children less than 12 years old , your doctor will adjust the dose for Niemann-Pick type C disease.

If you have a problem with your kidneys you may receive a lower starting dose. Your doctor may reduce your dose, e.g., to one capsule (100 mg) once or twice a day, if you suffer from diarrhoea when taking Miglustat Dipharma (see section 4). Your doctor will tell you how long your treatment will last.

Miglustat Dipharma can be taken with or without food. You should swallow the whole capsule with a glass of water.

If you take more Miglustat Dipharma than you should

If you take more capsules than you were told to, consult your doctor immediately. Miglustat has been used in clinical trials at doses ten times higher than the recommended dose: this caused decreases in white blood cells and other side effects similar to those described in section 4.

If you forget to take Miglustat Dipharma

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

If you stop taking Miglustat Dipharma

Do not stop taking Miglustat Dipharma 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.

Most serious side effects

Some patients have had tingling or numbness in the hands and feet (seen commonly). They could be signs of peripheral neuropathy, due to side effects of Miglustat Dipharma or they could be due to existing conditions. Your doctor will perform some tests before and during treatment with Miglustat Dipharma to assess this (see section 2).

If you do get any of these effects reported above, please seek medical advice from your doctor as soon as possible.

If you get a slight tremor , usually trembling hands, seek medical advice from your doctor as soon as possible. The tremor often disappears without needing to stop the treatment. Sometimes your doctor will need to reduce the dose or stop Miglustat Dipharma treatment to stop the tremor.

Very common side effects – may affect more than 1 in 10 people

The most common side effects are diarrhoea, flatulence (wind), abdominal (stomach) pain, weight loss and decreased appetite.

If you do lose some weight when you start treatment with Miglustat Dipharma don’t worry. People usually stop losing weight as treatment goes on.

Common side effects – may affect up to 1 in 10 people

Common side effects of treatment include headache, dizziness, paraesthesia (tingling or numbness), abnormal coordination, hypoaesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling sick), constipation and vomiting, swelling or discomfort in the abdomen (stomach) and thrombocytopenia (reduced levels of blood platelets). The neurological symptoms and thrombocytopenia could be due to the underlying disease.

Other possible side effects are muscular spasms or weakness, fatigue, chills and malaise, depression, difficulty sleeping, forgetfulness and reduced libido.

Most patients get one or more of these side effects mentioned above, usually at the start of treatment or at intervals during treatment. Most cases are mild and disappear quite quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of Miglustat Dipharma or recommend other medicines to help control side effects.

Reporting of side effects

If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. Y ou can also report side effects directly via 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. 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 which is stated on the blister and the carton after EXP. The expiry date refers to the last day of that month.

This medicinal product does not require any special storage conditions.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information

What Miglustat Dipharma contains

  • – The active substance is miglustat. Each hard capsule contains 100 mg miglustat.

  • – The other ingredients are magnesium stearate, gelatine, titanium dioxide (E171), printing ink

What Miglustat Dipharma looks like and contents of the pack

Miglustat Dipharma is a white opaque 100 mg capsule with “DPH02” printed in black on the top and “100” printed in black on the body. The capsules are presented in PCTFE/PVC and Aluminium blister, in a pack size of 84 capsules in non-perforated blisters and 84×1 capsules in perforated unit dose blisters.

Not all pack sizes may be marketed.

Marketing Authorisation Holder

Dipharma B.V.

Prins Bernhardplein 200

1097JB Amsterdam

Netherlands

Manufacturer

Doppel Farmaceutici S.r.l

Via Volturno 48

20089 Quinto de Stampi – Rozzano (MI)

Italy

This leaflet was last revised in {MM/YYYY}

Detailed information on this medicine is available on the European Medicines Agency web site:

24

The drug is classified in the ATC tree:

Frequently Asked Questions

What is Miglustat Dipharma used for?

Miglustat Dipharma is primarily used to treat certain types of Gaucher disease, a genetic disorder that affects how your body processes fats.

How does Miglustat work in the body?

Miglustat works by inhibiting an enzyme that breaks down certain fats, helping to reduce the buildup of these fats in the body.

What are the common side effects of Miglustat?

Common side effects may include diarrhea, stomach pain, nausea, and fatigue. Always consult your doctor if you experience persistent symptoms.

Can Miglustat be taken with other medications?

Before taking Miglustat with other medications, consult your healthcare provider to avoid potential interactions.

Is there a special diet I should follow while taking Miglustat?

While there isn't a specific diet for Miglustat, maintaining a balanced diet and staying hydrated can help manage side effects.

How long does it take to see results from Miglustat treatment?

Patients may start to notice improvements over several weeks or months; however, individual responses can vary.

What should I do if I miss a dose of Miglustat?

If you miss a dose, take it as soon as you remember. If it’s close to the time for your next dose, skip the missed dose and continue with your schedule.

Can Miglustat affect my liver function?

Miglustat can affect liver function in some patients; regular monitoring through blood tests is advised during treatment.

Is it safe to use Miglustat during pregnancy or breastfeeding?

Miglustat should only be used during pregnancy or breastfeeding if absolutely necessary. Consult your doctor for guidance.

What happens if I stop taking Miglustat suddenly?

Stopping Miglustat suddenly can lead to worsening symptoms of Gaucher disease. Consult your doctor before making any changes to your treatment plan.

Are there any long-term effects of taking Miglustat?

Long-term effects may include changes in weight and gastrointestinal symptoms. Regular check-ups are important to monitor health over time.

Can Miglustat be taken by children?

Miglustat is sometimes prescribed for children with Gaucher disease; however, dosage and monitoring must be carefully managed by a healthcare professional.

How should I store Miglustat Dipharma?

Store Miglustat at room temperature away from moisture and heat. Keep it out of reach of children.

What precautions should I take while on Miglustat treatment?

Regular blood tests are essential while taking Miglustat; inform your doctor about any new symptoms or health issues that arise.

Can lifestyle changes improve my condition while on Miglustat?

Yes, lifestyle changes such as regular exercise, healthy eating, and avoiding alcohol can potentially enhance your overall well-being while using Miglustat.

Is there any support available for patients using Miglustat?

Yes, many organizations offer support groups and resources for patients managing Gaucher disease. Your healthcare provider can help connect you with these resources.

What should I discuss with my doctor before starting Miglustat?

Discuss your medical history, current medications, potential side effects, and any concerns about treatment options with your doctor before starting.

Can I drink alcohol while taking Miglustat?

It's advisable to limit alcohol consumption while on Miglustat as it may exacerbate some side effects. Consult your healthcare provider for personalized advice.

How often will I need follow-up appointments while on Miglustat therapy?

Follow-up appointments may be scheduled every few months initially; however, the frequency will depend on your individual response and doctor’s recommendations.

Are there any online resources where I can learn more about Gaucher disease and Miglustat?

Yes, reputable health organizations like the National Gaucher Foundation provide valuable information regarding Gaucher disease and treatments like Miglustat.