Miglustat accord

Package leaflet: Information for the user

Miglustat Accord, 100 mg, hard capsules
Miglustat
Please read all of 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 further questions, ask your doctor or pharmacist.
• This medicine has been prescribed for a specific individual only. Do not pass it on to others. It may harm other people, even if their symptoms are the same.
• If you experience any side effects, including any not listed in this leaflet, tell your doctor or pharmacist. See section 4.

Contents of the leaflet

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

1. What Miglustat Accord is and what it is used for
Miglustat Accord contains an active substance called miglustat, which belongs to a group of medicines affecting metabolic processes. It is used to treat two conditions:

Miglustat Accord is used to treat mild to moderate type I Gaucher disease in adults.
In people with type I Gaucher disease, a substance called glucosylceramide is not removed from the body but accumulates in certain immune system cells. This leads to enlargement of the liver and spleen, blood abnormalities, and bone disease.
The standard treatment for type I Gaucher disease is enzyme replacement therapy. Miglustat Accord is used only in patients for whom enzyme replacement therapy is not suitable.

Miglustat Accord is also used to treat progressive neurological symptoms of Niemann-Pick disease type C in adults and children.
In people with Niemann-Pick disease type C, fats such as glycosphingolipids accumulate in brain cells. This may cause neurological problems, such as slow eye movements, balance and swallowing difficulties, memory problems, and seizures.

Miglustat Accord works by blocking an enzyme called "glucosylceramide synthase", which is responsible for the first step in the synthesis of most glycosphingolipids.

2. Important information before taking Miglustat Accord

When not to take Miglustat Accord

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

Warnings and precautions
Before starting Miglustat Accord, consult your doctor or pharmacist if:

• you have kidney disease,
• you have liver disease.

Your doctor will perform the following tests before and during treatment with Miglustat Accord:

• examination of nerves in hands and feet,
• measurement of vitamin B₁₂ levels,
• monitoring of growth in children and adolescents with Niemann-Pick disease type C,
• monitoring of platelet count.

These tests are performed because some patients may experience tingling or numbness in hands and feet, or weight loss during treatment with Miglustat Accord. These tests will help your doctor determine whether such symptoms are caused by the disease or other existing conditions, or whether they are side effects of Miglustat Accord (see details in section 4).

If you develop diarrhoea, your doctor may recommend dietary changes (to reduce intake of lactose and other carbohydrates such as sucrose (sugar)), taking Miglustat Accord between meals, or temporarily reducing the dose. In some cases, your doctor may prescribe an anti-diarrhoeal medicine, such as loperamide. Cases of Crohn's disease (inflammatory bowel disease) have been reported in patients with Niemann-Pick disease type C treated with Miglustat Accord. If diarrhoea persists despite these measures, or if other abdominal symptoms occur, consult your doctor. In such cases, your doctor may recommend further diagnostic tests to determine whether there is another cause for the symptoms.

Men should use effective contraception during treatment with Miglustat Accord and for 3 months after stopping the medicine.

Children and adolescents
Do not use this medicine in children and adolescents (under 18 years of age) with type I Gaucher disease, as it is not known whether the medicine is effective in treating this condition.

Miglustat Accord with other medicines
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 together with Miglustat Accord. Such medicines may reduce the amount of Miglustat Accord in your body.

Pregnancy, breastfeeding and fertility
You must not take Miglustat Accord if you are pregnant or planning to become pregnant. For further information, consult your doctor. Effective contraception must be used during treatment with Miglustat Accord. Do not breastfeed while taking Miglustat Accord.
Men should use effective contraception during treatment with Miglustat Accord and for 3 months after stopping the medicine.

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, consult your doctor or pharmacist before taking this medicine.

Driving and using machines
Miglustat Accord may cause dizziness. Do not drive, operate machinery, or use tools if you experience dizziness.

Miglustat Accord contains sodium
This medicine contains less than 1 mmol of sodium (23 mg) per capsule, i.e. it is considered "sodium-free".

3. How to take Miglustat Accord

This medicine should always be taken exactly as your doctor has instructed. If you are unsure, you should contact your doctor or pharmacist.

In type I Gaucher disease: The usual dose for adults is one capsule (100 mg) taken three times daily (in the morning, in the afternoon, and in the evening). This means that the maximum daily dose is three capsules (300 mg).

In Niemann-Pick disease type C: The usual dose for adults and adolescents (aged over 12 years) is two capsules (200 mg) taken three times daily (in the morning, in the afternoon, and in the evening). This means that the maximum daily dose is six capsules (600 mg).

For children under 12 years of age with Niemann-Pick disease type C, the doctor will adjust the dose accordingly.

If a patient has kidney problems, they may be given a lower starting dose. The doctor may reduce the dose, for example, to one capsule (100 mg) once or twice daily, especially if diarrhoea occurs during treatment with Miglustat Accord (see section 4). Your doctor will inform you about the duration of treatment.

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

Taking more Miglustat Accord than prescribed

If you take more capsules than recommended, contact your doctor immediately.

In clinical studies, miglustat has been administered at doses up to 3000 mg, which resulted in reduced white blood cell count and other adverse effects similar to those described in section 4.

If you miss a dose of Miglustat Accord

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

Stopping treatment with Miglustat Accord

Do not stop taking Miglustat Accord without consulting your doctor.

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

4. Possible adverse reactions

Like all medicines, this medicine can cause adverse reactions, although not everybody gets them.
Most serious adverse reactions:
Tingling or numbness in the hands and feet (frequently observed) has occurred in some patients.
These may be symptoms of peripheral neuropathy, either related to adverse effects of Miglustat Accord or to pre-existing diseases. To assess this, your doctor will perform certain tests before starting treatment and during treatment with Miglustat Accord (see section 2).
If any of these adverse reactions occur in the patient, the doctor should be informed immediately.
If the patient develops mild tremor, mainly tremor of the hands, the doctor should be informed immediately. Tremor often resolves without the need to discontinue treatment. Sometimes, however, it may be necessary to reduce the dose of Miglustat Accord or discontinue treatment with this medicine to control tremor.

Very common: (may occur in more than 1 in 10 people)
The most frequently occurring adverse reactions include: diarrhoea, flatulence (passing wind), abdominal (stomach) pain, weight loss and decreased appetite.
If the patient experiences weight loss after starting treatment with Miglustat Accord, there is no need to be concerned. This adverse reaction usually resolves with continued treatment.

Common: (may occur in up to 1 in 10 people)
Common adverse reactions associated with the use of the medicine include: headache, dizziness, paraesthesia (tingling or numbness), coordination disorders, hypoesthesia (reduced sense of touch), dyspepsia (heartburn), nausea, constipation and vomiting, abdominal distension or discomfort in the abdominal cavity (stomach), and thrombocytopenia (reduced platelet count). Neurological symptoms and thrombocytopenia may be caused by the underlying disease.

Other possible adverse reactions include: muscle cramps or weakness, fatigue, chills and malaise, depression, sleep disorders, memory impairment and decreased libido.
In most patients, one or more of these adverse reactions usually occur at the beginning of treatment or intermittently during treatment. Most cases are mild and resolve relatively quickly. If any of these adverse symptoms cause problems, contact the doctor, who may reduce the dose of Miglustat Accord or recommend other medicines to help manage the adverse reactions.

Reporting of adverse reactions
If any adverse reactions occur, including any adverse reactions not listed in this leaflet, inform a doctor or pharmacist. Adverse reactions can be reported directly to the Department for Monitoring Adverse Drug Reactions of the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Al. Jerozolimskie 181C
02-222 Warsaw
tel.: +48 22 49 21 301
fax: +48 22 49 21 309
website: https://smz.ezdrowie.gov.pl
Adverse reactions can also be reported to the marketing authorisation holder.
Reporting adverse reactions helps provide more information on the safety of the medicine.

5. How to store Miglustat Accord

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the carton after: EXP.
The expiry date refers to the last day of the stated month.
No special storage conditions are required for this medicine.
Medicines should not be disposed of via the sewage system or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help protect the environment.

6. Contents of the package and other information

What Miglustat Accord contains

• The active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
• The other ingredients are: sodium carboxymethyl starch (type A), povidone K30, magnesium stearate, gelatin, titanium dioxide (E171).

What Miglustat Accord looks like and contents of the pack
Miglustat Accord is a white, hard gelatin capsule filled with white to off-white granules, packed in PVC/PE/PCTFE/Aluminium blisters in a cardboard box.
Pack sizes: 14 x 1 or 84 x 1 capsules.
Not all pack sizes may be marketed.
Marketing Authorisation Holder
Accord Healthcare Polska Sp. z o.o.
Taśmowa Street 7
02-677 Warsaw
Tel: +48 22 577 28 00
Manufacturer
G.L. Pharma GmbH
Schlossplataz 1
8502 Lannach
Austria
This medicinal product is authorised in the European Economic Area member states under the following names: