100 mg hard capsules
miglustat
Read all of this leaflet carefully before you start taking this medicine. It contains information that is important to you.
- Save this information, 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 give it to others. It can harm them, even if they show signs of illness similar to yours.
- If you get any side effects , talk to your doctor or pharmacist. This also applies to any side effects not mentioned in this information. See section 4.
In this leaflet you will find information about:
1. What Miglustat Gen.Orph is and what it is used for
2. What you need to know before you use Miglustat Gen.Orph
3. How to take Miglustat Gen.Orph
4. Possible side effects
5. How Miglustat Gen.Orph should be kept
6. Contents of the package and other information
What Miglustat Gen.Orph is and what it is used for
Miglustat Gen.Orph contains the active substance miglustat, which belongs to a group of medicines that affect metabolism. It is used to treat two diseases:
- Miglustat Gen.Orph 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 Gen.Orph is used only if the patient is not considered suitable for treatment with enzyme replacement therapy.
- Miglustat Gen.Orph is also used to treat the growing neurological symptoms of Niemann-Pick type C in adults and children.
If you have Niemann-Pick 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 Gen.Orph works by inhibiting an enzyme called “glucosylceramide synthetase”, which is responsible for the first step in the production of most glycosphingolipids.
What you need to know before using Miglustat Gen.Orph
Do not take Miglustat Gen.Orph:
- 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 Gen.Orph
- 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 Gen.Orph:
- examination to check the nerves in your arms and legs
- measurement of vitamin B 12 ‑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 Gen.Orph. 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 Gen.Orph (for other 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 Gen.Orph 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 Gen.Orph 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 Gen.Orph
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 Gen.Orph. They can lower the amount of Miglustat Gen.Orph in your body.
Pregnancy, breastfeeding and fertility
You should not take Miglustat Gen.Orph if you are pregnant or planning to become pregnant. Your doctor can give you more information. You must use safe contraception when taking Miglustat Gen.Orph. Do not breastfeed while taking Miglustat Gen.Orph.
Male patients should use reliable contraception during treatment with Miglustat Gen.Orph 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 Gen.Orph may make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy.
How to take Miglustat Gen.Orph
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 capsule (100 mg) three times a day (morning, afternoon and evening). This means a maximum daily dose of three 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 maximum daily 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 one capsule (100 mg) once or twice a day if you suffer from diarrhea during treatment with Miglustat Gen.Orph (see section 4). Your doctor will tell you how long you will be treated.
Miglustat Gen.Orph can be taken with or without food. You should swallow the whole capsule with a glass of water.
If you take more Miglustat Gen.Orph than you should
Talk to your doctor immediately if you have taken more capsules than you should. Miglustat Gen.Orph 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 Gen.Orph
Take the next capsule at the usual time. Do not take a double dose to make up for a forgotten dose.
If you stop taking Miglustat Gen.Orph
Do not stop taking Miglustat Gen.Orph 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 Gen.Orph or due to pre-existing conditions. Your doctor will perform some checks before and during treatment with Miglustat Gen.Orph 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 Gen.Orph, to get rid of the tremors.
Very common – may affect more than 1 in 10 people
The most common side effects are diarrhea, gas, abdominal pain, weight loss, and decreased appetite.
Do not worry if you lose any weight when you start treatment with Miglustat Gen.Orph. You usually stop losing weight when the treatment continues.
Common – can affect up to 1 in 10 people
Common side effects of the treatment include headache, dizziness, paracentesis (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 one of Miglustat Gen.Orph or suggest other medications that can help reduce the side effects.
How to store Miglustat Gen.Orph
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.
This medicine does not require any special storage instructions.
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 100 mg.
- The other ingredients are:
- sodium starch glycolate (type A),
- povidone (K30),
- magnesium stearate.
- gelatine,
- titanium dioxide (E171).
What the medicine looks like and contents of the pack
Miglustat Gen.Orph 100 mg hard capsules are white opaque end and side, hard gelatin capsules, size 4, 14.0 mm long.
Pack size of 84 capsules in non-perforated blister packs and 84×1 capsules in perforated unit dose blister packs.
Not all pack sizes may be marketed.
Marketing Authorisation Holder:
Gen.Orph
185 Bureaux de la Colline
92213 Saint Cloud Cedex
France
Manufacturer:
Delpharm Reims
10 rue Colonel Charbonneaux
51100 Reims
France
or
Center Lab
ZA Granderaie
23000 Guéret
France
Contact the representative of the marketing authorization holder to find out more about this medicine:
| Belgium / Belgique / BelgienGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | LithuaniaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| Bulgaria Diakomers LODTel .: +359 2 807 50 00Email: diacommerce@diacommerce.bg | Luxembourg / LuxemburgGene. OrphTel / Tel .: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| Czech RepublicGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | HungaryGen.OrphTel .: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| DenmarkGen.OrphTel: +46 (0) 8 21 54 45email: pharmacovigilance.SE@propharmagroup.com | MaltaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| GermanyGen.OrphTel: +49 30 8560687897email: ph armacovigilance.DE@propharmagroup.com | The NetherlandsGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| EestiGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | NorwayGen.OrphTel: +46 (0) 8 21 54 45email: pharmacovigilance.SE@propharmagroup.com |
| Greece Gen.Orph:Ηλ: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | AustriaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| SpainPharma International SATel: +34 915 635 856e-mail:farmacovigilancia@pharmaintl.net | PolandGen.OrphTel .: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| FranceGen.OrphTel .: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | PortugalPharSolution Pharmaceutical Consulting, Lda.Tel: +351 21 464 32 25email: pharmacovigilance@pharsolution.com |
| CroatiaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | RomaniaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| IrelandGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | SloveniaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| IcelandGen.OrphPhone: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | Slovak RepublicGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |
| ItalyBiovalley Investments Partner SrlTel: +39 040 899 2219e.mail: info@biovalleyinvestmentspartner.it | Finland / FinlandGen.OrphPuh / Tel: +46 (0) 8 21 54 45email: pharmacovigilance.SE@propharmagroup.com |
| .Προς Gen.Orph:Ηλ: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | SwedenGen.OrphTel: +46 (0) 8 21 54 45email: pharmacovigilance.SE@propharmagroup.com |
| LatviaGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com | United KingdomGen.OrphTel: +33 (0) 1 47 71 04 50email: contact@gen-orph.com |