Prospecto: information for the user

Miglustat Accord 100 mg hard capsules EFG

Read this prospect carefully before starting to take this medicine, as it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you have any doubts, consult your doctor or pharmacist.
• This medicine has been prescribed only for you and should not be given to other people even if they have the same symptoms as you, as it may harm them.
• If you experience any adverse effects, consult your doctor or pharmacist, even if they are not listed in this prospect. See section 4.

1.Miglustat Accord and its use.

2.What you need to know before starting to take Miglustat Accord.

3.How to take Miglustat Accord.

4.Possible adverse effects.

5.Storage of Miglustat Accord.

6.Contents of the package and additional information.

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 Gaucher's disease type 1 in adults.

In Gaucher's disease type 1, your body cannot eliminate a substance called glucosylceramide. As a result, it accumulates in some cells of your immune system. This can lead to an enlarged liver and spleen, changes in the blood, and bone problems.

The usual treatment for Gaucher's disease type 1 consists of enzyme replacement therapy (ERT). Miglustat will only be used in cases where enzyme replacement therapy is not considered 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 accumulate in the cells of your brain. This can lead to neurological function alterations such as slow eye movements, balance, swallowing, memory, or seizures.

Miglustat acts by inhibiting the enzyme called glucosylceramidase synthase, responsible for the first step in the synthesis of most glycosphingolipids.

Do not take miglustat

• If you are allergic to miglustat or any of the other components of this medication (listed in section 6).

Warnings and precautions

Consult your doctor or pharmacist before starting to take 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 medication:

• Physical examination of the nerves in your arms and legs.
• Measurement of your vitamin B12 levels.
• Monitoring of growth in children or adolescents with Niemann-Pick type C disease.
• Monitoring of your 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 if these effects are due to your disease or existing conditions or are adverse 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 (sugar cane), or not take miglustat with food, or temporarily reduce the dose. In some cases, your doctor may prescribe medication to treat diarrhea, such as loperamide. Cases of Crohn's disease (an inflammatory disease affecting the intestine) have been reported in patients with Niemann-Pick type C disease treated with miglustat. Consult your doctor if diarrhea does not respond to these measures or if you experience any other abdominal discomfort. In this case, your doctor may decide to perform additional examinations.

It is recommended that male patients use reliable contraceptive methods during treatment with miglustat and for three months after completing treatment.

Children and adolescents

Do not administer this medication to children and adolescents (under 18 years of age) with Gaucher's disease type 1 because its effectiveness in this disease is unknown.

Other medications and Miglustat Accord

Inform your doctor or pharmacist if you are taking or have taken recently or may need to take any other medication.

Inform your doctor if you are taking medications containing 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 intend to become pregnant. For more information, consult your doctor or pharmacist. You will need to use an effective contraceptive method while taking miglustat. Do not continue breastfeeding while taking miglustat.

Male patients should use a reliable contraceptive method during treatment with miglustat, and for three months after completing treatment.

If you are pregnant or breastfeeding, or think you may be pregnant, or intend to become pregnant, consult your doctor or pharmacist before using this medication.

Driving and operating machinery

Miglustat may cause dizziness. Do not drive or operate tools or machinery if you feel dizzy.

Miglustat contains sodium

This medication contains less than 1 mmol of sodium (23 mg) per capsule; it is essentially "sodium-free".

Follow exactly the administration instructions for this medication as indicated by your doctor. If in doubt, consult your doctor or pharmacist again.

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

• For Niemann-Pick disease type C:

- In adults and adolescents (over 12 years old), the recommended dose is 2 capsules (200 mg) three times a day (morning, afternoon, and night). This represents a maximum daily dose of six capsules (600 mg).

- In children under 12 years old, your doctor will adjust the dose.

If you have kidney problems, your doctor may indicate a lower initial dose. Your doctor may reduce the miglustat dose to one capsule (100 mg) once or twice a day if diarrhea occurs during miglustat treatment (see section 4). Your doctor will indicate the duration of treatment.

Miglustat can be taken with or without food. Swallow the entire capsule with a glass of water.

If you take more miglustat than you should

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

In case of overdose or accidental ingestion, consult your doctor or pharmacist immediately or call the Toxicological Information Service, phone: 91 562 04 20, indicating the medication and the amount ingested.

If you forget to take miglustat

Take the next capsule when due. Do not take a double dose to compensate for the missed doses.

If you interrupt treatment with miglustat

Do not stop treatment with this medication without consulting your doctor.

If you have any other questions about the use of this medication, ask your doctor or pharmacist.

Like all medicines, this medicine may cause side effects, although not everyone will experience them.

More serious side effects:

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

Notify your doctor as soon as possible if you experience any of these side effects.

If youexperienceamild tremor, usuallyhand tremor,informyour doctor as soon as possible. Tremor often resolves without the need to interrupt treatment. In some cases, your doctor may need to reduce the dose and even interrupt treatment with miglustat to control the tremor.

Very common–may affect more than 1 in 10 patients

Among the most frequent side effects are diarrhea, flatulence (gas), abdominal pain (stomach), weight loss, and decreased appetite.

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

Common–may affect up to 1 in 10 patients

The common side effects are headache, dizziness, paresthesias (tingling or numbness), coordination disturbances, hypoaesthesia (reduced tactile sensation), dyspepsia (heartburn), nausea (feeling sick), constipation, and vomiting, abdominal swelling or discomfort, and thrombocytopenia (decreased platelet levels). Neurological symptoms and thrombocytopenia may be due to underlying disease.

Other possible side effects are 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 different times during treatment. Most are mild and resolve quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of miglustat or prescribe other medications to control side effects.

Reporting side effects:

If you experience any type of side effect, consult your doctor or pharmacist, even if it is a possible side effect that does not appear in this prospectus. You can also report them directly through the Spanish System of Pharmacovigilance of Medicines for Human Use: https://www.notificaram.es. By reporting side effects, you can contribute to providing more information on the safety of this medicine.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the packaging after “CAD”. The expiration date is the last day of the month indicated.

This medication does not require special storage conditions.

Medications should not be disposed of through drains or in the trash. Dispose of packaging and unused medications at the SIGRE collection point at the pharmacy. If in doubt, ask your pharmacist how to dispose of packaging and unused medications. By doing so, you will help protect the environment.

Composition of Miglustat Accord

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

The other components are sodium carboxymethyl starch from potato (type A), povidone, magnesium stearate, titanium dioxide (E171), gelatin.

Appearance of the product and contents of the packaging

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

Miglustat Accord is available in packaging 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

Responsible for manufacturing:

G.L. Pharma GmbH

Schlossplatz 1

8502 Lannach

Austria

Last review date of this leaflet:February 2025

The detailed information of this medicine is available on the website of the Spanish Agency of Medicines and Medical Devices (AEMPS) http://www.aemps.gob.es/.