Yargesa
ItalyTable of Contents
Package leaflet: Information for the user
Yargesa 100 mg hard capsules
miglustat
Please read 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 questions, ask your doctor or pharmacist.
- This medicine has been prescribed for you only. Do not give it to others, even if they have the same symptoms as yours, because it may be harmful.
- If you experience any adverse reactions, including those not listed in this leaflet, consult your doctor or pharmacist. See section 4.
Contents of this leaflet
- 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 conditions:
- Yargesa is used to treat mild to moderate type 1 Gaucher disease in adults.
Type 1 Gaucher disease is characterised by the accumulation in certain cells of the immune system of a substance called glucosylceramide that your body cannot eliminate. This accumulation may lead to enlargement of the liver and spleen, abnormalities in blood cells, and bone disease.
The standard treatment for type 1 Gaucher disease is enzyme replacement therapy. Yargesa is used only if a patient is considered unsuitable for treatment with enzyme replacement therapy.
- Yargesa is also used to treat progressive neurological symptoms in Niemann-Pick disease type C in adults and children.
If you have Niemann-Pick disease type C, fats (such as glycosphingolipids) build up within brain cells. This can cause neurological problems such as: slowed eye movements, impaired balance, swallowing difficulties, memory problems, and seizures.
Yargesa 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 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 taking Yargesa:
- if you have kidney disease;
- if you have liver disease.
Before and during treatment with Yargesa, your doctor will carry out the following checks:
- examination to assess the condition of the nerves in your upper and lower limbs;
- measurement of vitamin B levels;
- monitoring of growth in pediatric or adolescent patients affected by Niemann-Pick disease type C;
- monitoring of blood platelet count.
The reason these tests are performed is that, during treatment with Yargesa, some patients have experienced tingling or numbness in the hands and feet, or a decrease in body weight. These tests will help your doctor determine whether these effects are due to your underlying disease or other pre-existing conditions, or to side effects of this medicine (for further information see section 4).
If you have diarrhoea, your doctor may decide to: modify your diet to reduce lactose and carbohydrate intake such as sucrose (cane sugar); avoid taking Yargesa with food; temporarily reduce the dose of Yargesa. In some cases, your doctor may prescribe anti-diarrhoeal medicines such as loperamide. If your diarrhoea does not respond to the above measures or if you develop any other abdominal symptoms, consult your doctor. In such cases, your doctor may decide to perform further investigations.
Male patients must use reliable contraceptive methods during treatment with Yargesa and for 3 months after stopping the treatment.
Children and adolescents
Do not administer this medicine to children and adolescents (under 18 years of age) with type 1 Gaucher disease, as it is not known whether the medicine is effective for this condition.
Other medicines and Yargesa
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Inform your doctor if you are taking other medicines containing imiglucerase, which are sometimes used concomitantly with Yargesa. These may reduce your body's exposure to Yargesa.
Pregnancy, breastfeeding and fertility
Do not take Yargesa if you are pregnant or planning to become pregnant. Your doctor can provide you with further information. You must use an effective method of contraception during treatment with Yargesa. You must not breastfeed while taking Yargesa.
Male patients must continue to use reliable contraceptive methods during treatment with this medicine and for 3 months after stopping treatment.
If you are pregnant, suspect you may be pregnant, planning to become pregnant, or breastfeeding, consult your doctor or pharmacist before taking this medicine.
Driving and using machines
Yargesa may cause dizziness. If you feel dizzy, do not drive or operate tools or machinery.
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
Take this medicine exactly as prescribed by your doctor. If you have any doubts, consult your doctor or pharmacist.
Dosage
- For type 1 Gaucher disease: The usual dose for adults is one capsule (100 mg) three times a day (morning, afternoon, and evening), corresponding to a maximum of three capsules per day (300 mg).
- For Niemann-Pick disease type C: The usual dose for adults and adolescents (over 12 years of age) is 2 capsules (200 mg) three times a day (morning, afternoon, and evening), corresponding to a maximum daily dose of six capsules (600 mg). For children under 12 years of age, the doctor will adjust the dose appropriately for Niemann-Pick disease type C.
If you have kidney problems, you may initially be given a lower dose. Your doctor may decide to reduce the dose; for example, one capsule (100 mg) once or twice a day, especially if you experience diarrhea while taking Yargesa (see section 4). Your doctor will inform you of the duration of treatment.
How to remove the capsule:
- Tear along the perforated line
- Remove the foil at the position indicated by the arrows
- Press the product through the foil to release it
Yargesa can be taken on an empty or full stomach. 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. Miglustat has been used in clinical studies at doses up to 3000 mg, which caused reductions in white blood cell count and other adverse effects similar to those described in section 4.
If you forget to take Yargesa
Take the next capsule at your usual time. Do not take a double dose to make up for a missed dose.
If you stop taking Yargesa
Do not stop taking this medicine without first talking to your doctor.
If you have any questions about how to use this medicine, consult your doctor or pharmacist.
- Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody experiences them.
More serious side effects
Some patients have experienced tingling or numbness in the hands and feet (commonly observed). These may be signs of peripheral neuropathy, possibly due to side effects of Yargesa, or they may be related to the underlying condition. To assess these symptoms, your doctor will perform certain tests before and during treatment with Yargesa (see section 2).
If you experience any of these symptoms, consult your doctor as soon as possible.
If you experience mild tremor, usually hand tremor, consult your doctor as soon as possible. Tremor often resolves without discontinuing treatment. Sometimes your doctor may need to reduce the dose or stop treatment with Yargesa to resolve the tremor.
Very common side effects – may affect more than 1 in 10 people
The most common side effects include: diarrhea, flatulence (excessive intestinal gas), abdominal pain (stomach), weight loss, and reduced appetite.
If you lose weight when starting treatment with Yargesa, do not be concerned. Weight loss usually stops as treatment continues.
Common side effects – may affect up to 1 in 10 people
Common side effects of treatment include headache, dizziness, paresthesia (tingling or numbness), abnormal coordination, hypoesthesia (reduced touch sensitivity), dyspepsia (indigestion, heartburn), nausea, constipation, vomiting, abdominal bloating or discomfort (gastric), and thrombocytopenia (reduced platelet levels in the blood).
Neurological symptoms and thrombocytopenia may be due to the underlying disease.
Other possible side effects include: muscle spasms or weakness, fatigue, chills and malaise, depression, sleep disturbances, memory problems, and decreased libido.
Most patients experience one or more of these side effects, usually at the beginning of treatment or intermittently during treatment. These are mostly mild and disappear quickly. If any of these side effects cause discomfort, consult your doctor: they may reduce your dose of Yargesa or recommend other medicines to help manage the side effects.
Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, talk to your doctor or pharmacist. You can also report side effects directly via the national reporting system listed in Annex V. By reporting side effects, you help provide more information on the safety of this medicine.
- How to store Yargesa
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the blister and 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.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer in use. This will help protect the environment.
- Contents of the pack and other information
What Yargesa contains
- The active substance is miglustat. Each hard capsule contains 100 mg of miglustat.
- The other ingredients are:
Capsule contents: sodium starch glycolate (type A), povidone (K-29/32), magnesium stearate.
Capsule shell: gelatin, titanium dioxide (E171).
Printing ink: shellac, iron oxide black (E172), propylene glycol, and concentrated ammonia solution.
Description of Yargesa and contents of the pack
Yargesa is a white hard capsule consisting of a white opaque cap and body, with the number "708" printed in black on the body. The capsule size is 4 (14.3 mm x 5.3 mm).
The capsules are supplied in PVC and polychlorotrifluoroethylene (PCTFE) blisters, divided into unit doses and sealed with an aluminum foil.
Pack size: 84 x 1 hard capsules.
Marketing Authorization Holder
Piramal Critical Care B.V.
Rouboslaan 32 (ground floor)
2252 TR, Voorschoten
The Netherlands
Further information
More detailed information on this medicine is available on the website of the European Medicines Agency (http://www.ema.europa.eu).