Miglustat Accord 100 mg hard capsules EFG
Spain
Table of Contents
Package leaflet: Information for the user
Introduction
Package leaflet: information for the user
Miglustat Accord 100 mg hard capsules EFG
Read the entire leaflet carefully before you start taking this medicine, because it contains important information for you.
- Keep this leaflet, as 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 you should not give it to other people, even if they have the same symptoms as you, because it may harm them.
- If you experience any adverse effects, consult your doctor or pharmacist, even if they are adverse effects not listed in this leaflet. See section 4.
Contents of the leaflet
- What Miglustat Accord is and what it is used for.
- What you need to know before taking Miglustat Accord.
- How to take Miglustat Accord.
- Possible side effects.
- How to store Miglustat Accord.
- Contents of the pack and other information.
1. What Miglustat Accord is and what it is used for
Miglustat contains the active substance miglustat, which belongs to a group of medicines that affect metabolism. It is used to treat two disorders:
- Miglustat 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 cells, and problems affecting the bones.
The standard treatment for type 1 Gaucher disease is enzyme replacement therapy (ERT). Miglustat will only be used in cases where enzyme replacement therapy is considered unsuitable for the patient.
•Miglustat is also used to treat progressive neurological symptoms of Niemann-Pick disease type C in adults and children.
If you have Niemann-Pick disease type C, lipids such as glycosphingolipids may accumulate in the cells of your brain. This can lead to neurological impairments such as slowed eye movements, problems with balance, swallowing, memory, or seizures.
Miglustat 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 Accord
Do not take miglustat
- 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
- If you have kidney disease.
- If you have liver disease.
Your doctor will carry out the following tests before and during your treatment with this medicine:
- 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. 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 (see section 4 for more details).
If you experience diarrhoea, your doctor may advise you to modify your diet to reduce intake of lactose and carbohydrates such as sucrose (cane sugar), or to take miglustat without food, or to temporarily reduce the dose. In some cases, your doctor may prescribe medications to treat diarrhoea, such as loperamide. Cases of Crohn's disease (an inflammatory bowel disease) have been reported in patients with Niemann-Pick disease type C treated with miglustat. Consult your doctor if diarrhoea does not respond to these measures or if you develop 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 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 Accord
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. These may reduce the amount of miglustat in your body.
Pregnancy, breastfeeding and fertility
Do not take miglustat if you are pregnant or planning to become pregnant. For further information, consult your doctor or pharmacist. You should use an effective method of contraception while taking miglustat. Breastfeeding must not be continued while you are taking miglustat.
Male patients should use a reliable contraceptive method during treatment with miglustat and for three months after stopping treatment.
If you are pregnant or breastfeeding, think you may be pregnant or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.
Driving and using machines
Miglustat may cause dizziness. Do not drive or operate tools or machinery if you feel dizzy.
Miglustat contains sodium
This medicine contains less than 1 mmol of sodium (23 mg) per capsule; this is essentially “sodium-free”.
3. How to take Miglustat Accord
Follow exactly the instructions given by your doctor for taking this medicine. If in doubt, consult your doctor or pharmacist again.
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For type 1 Gaucher disease: in adults, the recommended dose is one capsule (100 mg) three times a day (morning, afternoon, evening), for a maximum daily dose of three capsules (300 mg).
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For Niemann-Pick disease type C:
-In adults and adolescents (over 12 years of age), the recommended dose is 2 capsules (200 mg) three times a day (morning, afternoon, and evening), for a maximum daily dose of six capsules (600 mg).
-In children under 12 years of age, your doctor will adjust the dose.
If you have kidney problems, your doctor may prescribe a lower starting dose. Your doctor may reduce the dose of miglustat to one capsule (100 mg) once or twice a day if you develop diarrhea during treatment with miglustat (see section 4). Your doctor will advise you on the duration of treatment.
Miglustat may be taken with or without food. The capsule should be swallowed whole with a glass of water.
If you take more miglustat 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 side effects similar to those described in section 4.
In case of overdose or accidental ingestion, contact your doctor or pharmacist immediately or call the Toxicology Information Service at telephone number: 91 562 04 20, indicating the medicine and the amount ingested.
If you forget to take miglustat
Take the next capsule at the usual time. Do not take a double dose to make up for forgotten doses.
If you stop taking miglustat
Do not stop treatment with this medicine 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 may cause adverse effects, although not everyone experiences them.
More serious adverse effects:
Some patients have experienced tingling or numbness in the hands and feet (frequent). These could be signs of peripheral neuropathy due to side effects of miglustat, or could be due to pre-existing diseases. Your doctor will perform tests before and during treatment with miglustat to assess your condition (see section 2).
If you experience any of these effects, inform your doctor as soon as possible.
If you experience a slight tremor, usually tremor in the hands, inform your doctor as soon as possible. Often, the tremor resolves without the need to interrupt treatment. Occasionally, your doctor may need to reduce the dose or even discontinue treatment with miglustat to control the tremor.
Very common – may affect more than 1 in 10 patients
The most common adverse effects include diarrhoea, flatulence (gas), abdominal pain (stomach pain), weight loss, and decreased appetite.
If you lose some weight when starting treatment with miglustat, do not worry; usually people stop losing weight as treatment continues.
Common – may affect up to 1 in 10 patients
Common adverse effects are 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 feeling unwell, depression, difficulty sleeping, forgetfulness, and decreased libido.
Most patients notice one or more of these side 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. They may reduce your dose of miglustat or prescribe other medicines to manage the side effects.
Reporting of adverse effects:
If you experience any type of adverse effect, consult your doctor or pharmacist, even if it is a possible adverse effect not listed in this leaflet. You can also report them directly via the Spanish Pharmacovigilance System for Human Medicines: https://www.notificaram.es. By reporting adverse effects, you can help provide more information on the safety of this medicine.
5. Storage of Miglustat Accord
Keep this medicine out of sight and reach of children.
Do not use this medicine after the expiry date stated on the packaging after "EXP". The expiry date is 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. Dispose of empty containers and unused medicines at the SIGRE collection point at your pharmacy. If you are in doubt, ask your pharmacist how to dispose of containers and medicines you no longer need. This will help protect the environment.
6. Contents of the pack and other information
Composition of Miglustat Accord
The active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
The other components are sodium carboxymethyl starch (potato type A), povidone, magnesium stearate, titanium dioxide (E171), gelatin.
Appearance of the product and contents of the pack
White, hard gelatin capsule filled with a homogeneous white to off-white granule, in Aclar-Alu (PVC/PE/PCTFE-Alu) blister pack.
Miglustat Accord is available in pack sizes of 84 hard capsules.
Marketing Authorization Holder:
Accord Healthcare S.L.U.
World Trade Center
Moll de Barcelona s/n, Edifici Est, 6th floor,
08039 Barcelona
Spain
Manufacturer:
G.L. Pharma GmbH
Schlossplatz 1
8502 Lannach
Austria
Date of the most recent review of this leaflet: February 2025
Detailed information on this medicine is available on the website of the Spanish Agency for Medicines and Health Products (AEMPS) http://www.aemps.gob.es/.