100 mg hard capsules
miglustat
What Miglustat Bluefish is and what it is used for
Miglustat Bluefish contains the active substance miglustat, which belongs to a group of medicines that affect metabolism. It is used to treat two diseases.
Miglustat Bluefish is used to treat mild to moderate Gaucher type 1 disease in adults.
For Gaucher type 1 disease, a substance called glucosylceramide is not removed from your body. It begins to be stored in certain cells that belong to the body’s immune system. This can lead to liver and spleen enlargement, changes in the blood, and skeletal disease.
Gaucher’s type 1 disease is normally treated with enzyme replacement therapy. Miglustat Bluefish is used only if the patient is not considered suitable for treatment with enzyme replacement therapy.
Miglustat Bluefish is also used to treat the growing neurological symptoms of Niemann-Pick type C in adults and children
If you have Niemann-Pick type C disease, fats, such as glycosphingolipids, are stored in your brain cells. This can lead to disturbances in neurological functions such as slow eye movements, balance, swallowing ability, memory, and to seizures.
Miglustat Bluefish acts by inhibiting an enzyme called “glucosylceramide synthetase”, which is responsible for the first step in the production of most glycosphingolipids.
Miglustat contained in Miglustat Bluefish may also be approved for the treatment of other diseases not mentioned in this leaflet. Ask your doctor, pharmacist, or other healthcare professionals if you have any further questions, and always follow their instructions.
What you need to know before you take Miglustat Bluefish
Do not take Miglustat Bluefish
- if you are allergic to miglustat or any of the other ingredients of this medicine (listed in section 6).
Warnings and cautions
Talk to your doctor or pharmacist before taking Miglustat Bluefish
- if you have any kidney disease
- if you have any liver disease
Your doctor will perform the following checks before and during treatment with Miglustat Bluefish:
- examination to check the nerves in your arms and legs
- measurement of vitamin B12 value
- monitor your growth if you are a child or adolescent with Niemann-Pick type C disease
- check the number of platelets
The reason for these controls is that some patients have had tingling or numbness in their hands and feet or lost weight during treatment with Miglustat Bluefish. The checks make it easier for the doctor to determine if these problems are due to your illness or something you have had in the past, or if they are due to side effects of Miglustat Bluefish (for more information see section 4).
If you get diarrhea, your doctor may ask you to change your diet so that you reduce your intake of milk sugar, and carbohydrates such as sucrose (regular sugar). Your doctor may also ask you not to take Miglustat Bluefish with food. In some cases, your doctor may temporarily lower your dose or prescribe a medicine for diarrhea (such as loperamide). If your diarrhea does not respond to these measures, or if you have any other stomach problems, consult your doctor. In this case, your doctor may decide on further investigations.
Male patients should use reliable contraception during treatment with Miglustat Bluefish and for 3 months after stopping treatment.
Children and young people
Do not give this medicine to children and adolescents (under 18 years of age) with Gaucher type 1 disease because it is not known if it works against that disease.
Other medicines and Miglustat Bluefish
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Miglustat Bluefish. They can lower the amount of Miglustat Bluefish in your body.
Pregnancy, breastfeeding and fertility
You should not take Miglustat Bluefish if you are pregnant or planning to become pregnant. Your doctor can give you more information. You must use safe contraception when taking Miglustat Bluefish. Do not breastfeed while taking Miglustat Bluefish.
Male patients should use reliable contraception during treatment with Miglustat Bluefish and 3 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 Bluefish can make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy.
You are responsible for assessing whether you are fit to drive a motor vehicle or perform work that requires sharpened attention. One of the factors that can affect your ability in these respects is the use of drugs due to their effects and/or side effects. The description of these effects and side effects can be found in other sections. Read all the information in this leaflet for guidance. If you are not sure, talk to your doctor or pharmacist.
Miglustat Bluefish contains sodium
This medicine contains less than 1 mmol (23 mg) sodium per 100 mg capsules, ie essentially ‘sodium-free’.
How to take Miglustat Bluefish
Always take this medicine exactly as your doctor has told you. Ask your doctor or pharmacist if you are unsure.
- For Gaucher disease type 1: for adult patients, the usual dose is one hard capsule (100 mg) three times a day (morning, afternoon and evening). This means a maximum daily dose of three hard capsules (300 mg).
- For Niemann-Pick type C disease: For adults and adolescents (over 12 years of age), the usual dose is one two capsules (200 mg) three times a day (morning, afternoon and evening). This means a daily maximum dose of 6 capsules (600 mg).
For children under the age of 12 who have Niemann-Pick type C disease, your doctor will adjust the dose one.
If you have kidney problems, you may receive a lower starting dose. Your doctor may reduce your dose e.g. to a hard capsule (100 mg) once or twice a day if you suffer from diarrhea during treatment with Miglustat Bluefish (see section 4). Your doctor will tell you how long you will be treated.
Miglustat Bluefish can be taken with or without food. You should swallow the whole capsule with a glass of water.
If you take more Miglustat Bluefish than you should
Miglustat Bluefish has been used in clinical trials at doses ten times higher than the recommended dose: this caused a decrease in the number of white blood cells and other side effects similar to those described in section 4.
If you forget to take Miglustat Bluefish
Take the next hard capsule at the usual time. Do not take a double dose to make up for a forgotten dose.
If you stop taking Miglustat Bluefish
Do not stop taking Miglustat Bluefish without talking to your doctor.
If you have any further questions on the use of this product, ask your doctor or pharmacist.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Most serious side effects:
Some patients have experienced tingling or numbness in the hands and feet (common). This may be a sign of peripheral neuropathy due to side effects of Miglustat Bluefish or due to pre-existing conditions. Your doctor will perform some checks before and during treatment with Miglustat Bluefish to evaluate this (see section 2).
If you experience any of these symptoms, consult your doctor as soon as possible.
If you experience mild tremors, usually in your hands, consult your doctor as soon as possible. These tremors usually disappear without the need to interrupt treatment. Sometimes your doctor will need to reduce the dose one or discontinue treatment with Miglustat Bluefish for you to get rid of the tremors.
Very common (may affect more than 1 user in 10):
The most common side effects are diarrhea, gas, abdominal (stomach) pain, weight loss, and decreased appetite. Do not worry if you lose any weight when starting treatment with Miglustat Bluefish. You usually stop losing weight when the treatment continues.
Common (may affect up to 1 in 10 people):
Common side effects of treatment include headache, dizziness, paresthesia (tingling or numbness), abnormal coordination, hypoesthesia (decreased sensation), acid regurgitation (heartburn), nausea, constipation and vomiting, swelling or discomfort from the abdomen (stomach), and thrombocytopenia (decreased platelet level ). The neurological symptoms and thrombocytopenia may be caused by the underlying disease.
Other possible side effects are muscle spasms or weakness, fatigue, chills and general malaise, depression, difficulty sleeping, forgetfulness, and decreased libido.
Most patients experience one or more of these side effects, usually at the beginning of treatment or periodically during treatment. In most cases, the side effects are mild and disappear fairly quickly. If any of these side effects occur, consult your doctor. He or she can then reduce the dose of Miglustat Bluefish or suggest other medications that can help reduce the side effects.
How to store Miglustat Bluefish
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 expiration date is the last day of the specified month.
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 to protect the environment.
Contents of the pack and other information
Content declaration
The active substance is miglustat. Each capsule contains 100 mg miglustat.
Other ingredients are:
Capsule contents: sodium starch glycolate type A, povidone, magnesium stearate
Capsule shell: titanium dioxide (E171), gelatin
What the medicine looks like and contents of the pack
White hard gelatin capsules, filled with homogeneous white to off-white granules in perforated Aclar-Alu blisters (PVC / PE / PCTFE-Alu blisters).
Miglustat Bluefish 100 mg hard capsules are available in packs of 84 hard capsules.
Marketing Authorisation Holder
Manufacturer
GL Pharma GmbH
Schlossplatz 1, Lannach 8502
Austria
This medicinal product is authorized under the European Economic Area under the names:
| Germany | Miglustat Bluefish 100 mg Heart Capsule |
| Denmark | Miglustat Bluefish |
| France | Miglustat Bluefish 100 mg gélules |
| Norway | Miglustat Bluefish 100 mg hard capsules |
| Sweden | Miglustat Bluefish 100 mg hard capsules |