MIGLUSTAT ACCORD 100 mg HARD CAPSULES

Introduction

Package Leaflet: Information for the User

Miglustat Accord 100 mg Hard Capsules EFG

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.

Contents of the pack

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

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

Miglustat contains the active substance miglustat, which belongs to a group of medicines that have an effect on 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 break down a substance called glucosylceramide. As a result, it builds up in some cells of your immune system. This can lead to an enlarged liver and spleen, changes in the blood, and effects on the bones.

The usual treatment for type 1 Gaucher disease is enzyme replacement therapy (ERT). Miglustat will only be used in cases where enzyme replacement therapy is not suitable for the patient.

• Miglustat is also used to treat the 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 can build up in the cells of your brain. This can lead to problems with neurological functions such as slow eye movements, balance, swallowing, memory, or seizures.

Miglustat 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 Accord

Do not take miglustat

• 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 you start taking miglustat

• If you have kidney disease.
• If you have liver disease.

Your doctor will perform the following tests before and during your treatment with this medicine:

• Physical examination of the nerves in your 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 the blood.

These tests are necessary because some patients have reported symptoms such as tingling or numbness in hands and feet, or weight loss during treatment with miglustat. The tests will help your doctor determine if these effects are due to your disease or existing conditions or are side effects of miglustat (see section 4 for more details).

If you have diarrhea, your doctor may ask you to modify your diet to reduce lactose and carbohydrate intake, such as sucrose (cane sugar), or not to take miglustat with food, or to temporarily reduce the dose. In some cases, your doctor may prescribe medications to treat diarrhea, such as loperamide. There have been reports of Crohn's disease (an inflammatory disease that affects the intestine) in patients with Niemann-Pick disease type C treated with miglustat. If diarrhea does not respond to these measures or if you experience any other abdominal discomfort, talk to your doctor. In this case, your doctor may decide to perform additional tests.

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 because it is not known if it works in this disease.

Other medicines and Miglustat Accord

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

Tell your doctor if you are taking medicines that contain imiglucerase, which are sometimes used at the same time as miglustat. They may decrease 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 more information, talk to your doctor or pharmacist. You must use an effective contraceptive method while taking miglustat. You should not breastfeed while 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 have a baby, ask your doctor or pharmacist for advice before taking this medicine.

Driving and using machines

Miglustat may cause dizziness. Do not drive or use tools or machines 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 the instructions for administration of this medicine exactly as told by your doctor. If you are unsure, talk to your doctor or pharmacist again.

• For type 1 Gaucher disease:in adults, the recommended dose is one capsule (100 mg) three times a day (morning, afternoon, evening), which is a maximum daily dose of three capsules (300 mg).

• For Niemann-Pick disease type C:

• In adults and adolescents (over 12 years), the recommended dose is 2 capsules (200 mg) three times a day (morning, afternoon, and evening). This is a maximum daily dose of six capsules (600 mg).
• In children under 12 years, your doctor will adjust the dose.

In case of kidney problems, your doctor may prescribe a lower initial dose. It is possible that your doctor will reduce the dose of miglustat to one capsule (100 mg) one or two times a day if you experience diarrhea during treatment with miglustat (see section 4). Your doctor will tell you how long to take the treatment.

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

If you take more miglustat than you should

If you take more capsules than you were told, talk to your doctor immediately. In clinical trials, miglustat has been used at doses of up to 3000 mg: this caused a reduction in blood leukocytes and other side effects similar to those described in section 4.

In case of overdose or accidental ingestion, talk to your doctor or pharmacist immediately or call the Toxicology Information Service, phone: 91 562 04 20, indicating the medicine and the amount taken.

If you forget to take miglustat

Take the next capsule when it is due. Do not take a double dose to make up for forgotten doses.

If you stop taking miglustat

Do not stop taking this medicine 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.

More serious side effects:

Some patients have reported tingling or numbness in hands and feet (this happens frequently).These could be signs of peripheral neuropathy, due to side effects of miglustat or could be due to existing diseases. Your doctor will perform tests before and during treatment with miglustat to assess your case (see section 2).

If you get any of these side effects, tell your doctor as soon as possible.

If you experiencea mild tremor, usually tremor in the hands, tellyour doctor as soon as possible. Often the tremor resolves without needing to stop treatment. In some cases, your doctor may need to reduce the dose and even stop treatment with miglustat to control the tremor.

Very common– may affect more than 1 in 10 people

The most common side effects include diarrhea, flatulence (gas), abdominal pain (stomach pain), weight loss, and decreased appetite.

If you lose some weight when you start taking miglustat, do not worry, people usually stop losing weight with continued treatment.

Common– may affect up to 1 in 10 people

Common side effects are headache, dizziness, paresthesia (tingling or numbness), coordination problems, hypoesthesia (reduced sensation to touch), dyspepsia (indigestion), nausea (feeling sick), constipation, and vomiting, abdominal swelling or discomfort (stomach) and thrombocytopenia (low blood platelet count). Neurological symptoms and thrombocytopenia may be due to the underlying disease.

Other possible side effects are muscle spasms or weakness, fatigue, chills, and feeling unwell, depression, difficulty sleeping, memory loss, and decreased libido.

Most patients notice one or more of these side effects, usually when starting treatment or at different times during treatment. Most are mild and resolve quickly. If any of these side effects bother you, talk to your doctor. They may reduce the dose of miglustat or prescribe other medicines to control the 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. You can also report side effects directly to the Spanish Medicines and Healthcare Products Agency (AEMPS) via their website: https://www.notificaram.es. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Miglustat Accord

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 carton after "EXP". The expiry date refers to the last day of that month.

This medicine does not require any special storage conditions.

Medicines should not be disposed of via wastewater 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 pack and other information

Composition of Miglustat Accord

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

The other ingredients are potato starch, povidone, magnesium stearate, titanium dioxide (E171), and gelatin.

Appearance and packaging

White hard gelatin capsule filled with a homogeneous white to off-white granulate in Aclar-Alu (PVC/PE/PCTFE-Alu) blister packs.

Miglustat Accord is available in pack sizes of 84 hard capsules.

Marketing authorisation holder:

Accord Healthcare S.L.U.

World Trade Center

Moll de Barcelona s/n, Edifici Est, 6ª planta,

08039 Barcelona

Spain

Manufacturer:

G.L. Pharma GmbH

Schlossplatz 1

8502 Lannach

Austria

Date of last revision of this leaflet:February 2025

Detailed information on this medicine is available on the website of the Spanish Agency for Medicines and Healthcare Products (AEMPS) http://www.aemps.gob.es/.