Yargesa 100 mg hard capsules EFG
Spain
Table of Contents
Package leaflet: Information for the user
Introduction
Package leaflet: information for the user
Yargesa 100 mg hard capsules
miglustat
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 must not be given to other people, even if they have the same symptoms as you, because it may harm them.
- If you experience any adverse reactions, consult your doctor or pharmacist, even if they are adverse reactions not listed in this leaflet. See section 4.
Leaflet contents:
- What Yargesa is and what it is used for.
- What you need to know before taking Yargesa.
- How to take Yargesa.
- Possible side effects.
- How to store Yargesa.
- Contents of the pack and other information.
1. What Yargesa is and what it is used for
Yargesa contains the active substance miglustat, which belongs to a group of medicines that affect metabolism. It is used to treat two disorders:
- Yargesa 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, it accumulates in certain cells of your immune system. This may cause your liver and spleen to become enlarged, changes in blood parameters, and affect your bones.
The standard treatment for type 1 Gaucher disease is enzyme replacement therapy (ERT). Yargesa will only be used in cases where enzyme replacement therapy is considered unsuitable for the patient.
- Yargesa 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 impairment, including slowed eye movements, problems with balance, swallowing, memory, or seizures.
Yargesa works by inhibiting the enzyme known as glucosylceramide synthase, which is responsible for the first step in the synthesis of most glycosphingolipids.
2. What you need to know before taking Yargesa
Do not take Yargesa
- 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 starting to take Yargesa
- if you have kidney disease
- if you have liver disease
Your doctor will carry out the following tests before and during your treatment with Yargesa:
- 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 blood platelet count
These tests are necessary because some patients have reported symptoms such as tingling or numbness in the hands and feet, or a reduction in body weight during treatment with this medicine. The tests will help your doctor determine whether these effects are due to your disease or pre-existing conditions, or are adverse effects of Yargesa (see section 4 for more details).
If you have diarrhoea, your doctor may advise you to modify your diet to reduce intake of lactose and carbohydrates such as sucrose (cane sugar), or not to take Yargesa with food, or to temporarily reduce the dose. In some cases, your doctor may prescribe medication to treat diarrhoea, such as loperamide. Cases of Crohn's disease (an inflammatory disease affecting the intestine) have been reported in patients with Niemann-Pick disease type C treated with Yargesa. Consult your doctor if diarrhoea does not respond to these measures or if you experience any other abdominal discomfort. In such cases, your doctor may decide to carry out additional investigations to determine whether there is another cause of your symptoms.
Male patients are advised to use reliable contraceptive methods during treatment with Yargesa 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 unknown whether it is effective in this condition.
Taking Yargesa with other medicines
Tell your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines.
Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Yargesa. These may reduce the amount of Yargesa in your body.
Pregnancy, breast-feeding and fertility
Do not take Yargesa if you are pregnant or planning to become pregnant. For more information, consult your doctor or pharmacist. You should use an effective method of contraception while taking Yargesa. You must not breast-feed while taking Yargesa.
Male patients should use a reliable method of contraception during treatment with this medicine, and for three months after stopping treatment.
If you are pregnant or breast-feeding, think you may be pregnant or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.
Driving and using machines
Yargesa may cause dizziness. Do not drive or operate tools or machinery if you feel dizzy.
Yargesa contains sodium
This medicine contains less than 1 mmol of sodium (23 mg) per capsule; i.e., essentially "sodium-free".
3. How to take Yargesa
Follow exactly the instructions for administering this medicine as given by your doctor. If in doubt, consult your doctor or pharmacist again.
Dosage
-
For Gaucher disease type 1: In adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon, evening), representing a maximum daily dose of three capsules (300 mg).
-
For Niemann-Pick disease type C: In adults and adolescents (over 12 years of age), the usual dose is two capsules (200 mg) three times a day (morning, afternoon, and evening). This corresponds to a maximum daily dose of six capsules (600 mg).
For children under 12 years of age, your doctor will adjust the dose for Niemann-Pick disease type C.
In case of kidney problems, your doctor may prescribe you a lower initial dose. Your doctor may reduce the dose of Yargesa to one capsule (100 mg) once or twice a day if diarrhea occurs during treatment with Yargesa (see section 4). Your doctor will advise you on the duration of treatment.
To remove the capsule:
- Separate along the perforated line
- Remove the paper as indicated by the arrows
- Push the capsule through the aluminum foil
Yargesa can be taken with or without food. The capsule must be swallowed whole with a glass of water.
If you take more Yargesa 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.
If you forget to take Yargesa
Take the next capsule at the scheduled time. Do not take a double dose to make up for missed doses.
If you stop taking Yargesa
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 can cause adverse effects, although not everyone will experience them.
More serious adverse effects
Some patients have experienced tingling or numbness in the hands and feet (this is seen frequently). These could be signs of peripheral neuropathy due to side effects of Yargesa, or they could be due to pre-existing diseases. Your doctor will carry out tests before and during treatment with Yargesa 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 trembling of 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 Yargesa to control the tremor.
Very common – may affect more than 1 in 10 people
Among the most common adverse effects are diarrhoea, flatulence (gas), abdominal pain (stomach pain), weight loss and decreased appetite.
If you lose some weight when starting treatment with Yargesa, do not be concerned; usually people stop losing weight as treatment continues.
Common – may affect up to 1 in 10 people
Common adverse effects include headache, dizziness, paraesthesia (tingling or numbness), coordination disturbances, 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, difficulty sleeping, forgetfulness and decreased libido.
Most patients notice one or more of these types of side effects, usually when starting treatment or at different times during treatment. Most are mild and resolve quite quickly. If any of these adverse effects cause you problems, consult your doctor. He or she may reduce your dose of Yargesa or prescribe other medicines to manage the side effects.
Reporting of adverse effects
If you experience any adverse effects, talk to your doctor or pharmacist, even if they are adverse effects not listed in this leaflet. You can also report them directly through the national reporting system listed in Annex V. By reporting adverse effects, you can help provide more information on the safety of this medicine.
5. Yargesa Storage
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the blister pack and outer carton 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. Ask your pharmacist how to dispose of medicines and packaging that you no longer need. This will help protect the environment.
6. Contents of the pack and other information
Composition of Yargesa
- The active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
- The other components are:
Capsule contents: sodium starch glycolate (type A), povidone (K29/32), magnesium stearate,
Capsule shell: gelatin, titanium dioxide (E171),
Printing ink: enamel, black iron oxide (E172), propylene glycol, concentrated ammonia solution
Appearance of the product and contents of the pack
Yargesa is a white, opaque hard capsule consisting of a body and a cap, both white, with "708" printed in black on the body. The capsule size is 4 (14.3 mm x 5.3 mm). The capsules are presented in a PVC and polychlorotrifluoroethylene (PCTFE) blister sealed with an aluminum foil.
Pack size: 84 x 1 hard capsules.
Marketing Authorization Holder and Manufacturer
Piramal Critical Care B.V.
Rouboslaan 32 (ground floor)
2252 TR, Voorschoten
The Netherlands
Date of the most recent review of this leaflet: <{MM/YYYY}><{month YYYY}>.
Other sources of information
Detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu.