Orphacol - patient leaflet, side effects, dosage

- If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

What is in this leaflet :

• 1. What Orphacol is and what it is used for

• 2. What you need to know before you take Orphacol

• 3. How to take Orphacol

• 4. Possible side effects

• 5. How to store Orphacol

• 6. Contents of the pack and other information

1. What Orphacol is and what it is used for

Orphacol contains cholic acid, a bile acid which is normally produced by the liver. Certain medical conditions are caused by defects in bile acid production and Orphacol is used to treat infants from one month to 2 years of age, children, adolescents and adults with these medical conditions. The cholic acid contained in Orphacol replaces the bile acids that are missing due to the defect in bile acid production.

2. What you need to know before you take Orphacol

Do not take Orphacol

• – if you are allergic to cholic acid or any of the other ingredients of this medicine (listed in section 6).

• – if you are taking phenobarbital, a medicine to treat epilepsy.

Warnings and precautions

During your treatment, your doctor will carry out various blood and urine tests at different time to see how your body is handling this medicine and to help work out the dose that you need. More frequent tests will be needed if you are growing fast, if you are ill (if you have e.g. liver problems), or if you are pregnant.

Other medicines and Orphacol

Tell your doctor or pharmacist if you are taking, have recently taken any or might take other medicines.

Some medicines used to lower cholesterol levels, so-called bile acid sequestrants (cholestyramine, colestipol, colesevelam), and medicines to treat heartburn that contain aluminium may lessen the effect of

Orphacol. If you take these medicines, take Orphacol at least 5 hours before or at least 5 hours after taking these other medicines.

Ciclosporin (a medicine used to suppress the immune system) may also change the effect of Orphacol. Please tell your doctor if you are taking ciclosporin.

Pregnancy and breast-feeding

Please consult your doctor if you plan to become pregnant. Take a pregnancy test as soon as you suspect you may be pregnant. It is very important to continue taking Orphacol during pregnancy.

If you become pregnant during treatment with Orphacol, your doctor will decide which treatment and dose is best in your situation. As a precaution, you and your unborn child should be closely monitored during pregnancy.

Orphacol can be used during breast-feeding. Tell your doctor if you plan to breast-feed or are breastfeeding before you take Orphacol.

Ask your doctor or pharmacist for advice before taking any medicine.

Driving and using machines

Orphacol is not expected to have an effect on the ability to drive and use machines.

Orphacol contains lactose

Orphacol contains a certain sugar (lactose monohydrate). If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor before taking Orphacol.

3. How to take Orphacol

Always take Orphacol exactly as your doctor has told you. Check with your doctor if you are not sure.

The usual starting dose is 5 to 15 mg per kilogram body weight every day in infants, children, adolescents and adults.

Before treatment your doctor will make an assessment from your laboratory tests to determine the correct dose for you. The dose will then be further adjusted by your doctor depending on your body’s response.

Orphacol capsules are taken by mouth with a meal at approximately the same time each day, in the morning and/or evening. Taking Orphacol at regular times with a meal will help you remember to take this medicine, and may help your body to better take it up. Capsules must be swallowed whole with water. Do not chew.

If your doctor has prescribed a dose which requires you to take more than one capsule per day, you and your doctor can decide how this will be taken during the day. You may for example take one capsule in the morning and one in the evening. This way, you will have to take fewer capsules at once.

Use in children

For babies and children who cannot swallow capsules, the capsule can be opened and its contents added to baby formula or apple/orange or apple/apricot juice adapted for small children.

If you take more Orphacol than you should

If you have taken more Orphacol than you should, contact your doctor as soon as possible. He will assess your laboratory test results and advise you when you should resume your treatment with your normal dose.

If you forget to take Orphacol

Take your next dose when you would normally take it. Do not take a double dose to make up for a forgotten dose.

If you stop taking Orphacol

There is a risk of permanently damaging your liver if you stop taking Orphacol. You should never stop taking Orphacol unless your doctor advises you to do so.

If you have any further questions on the use of this medicine, ask your doctor.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Several patients have experienced itching and/or diarrhoea, however, it is not known how likely this is to occur (frequency cannot be estimated from the available data). If itching and/or diarrhoea last for more than three days, tell your doctor.

For several patients an increase of liver enzymes (serum transaminases) have been reported during the treatment with Orphacol (frequency cannot be estimated from the available data). Your doctor will decide what to do if this happens to you.

Gallstones have been reported after long-term therapy with Orphacol.

Reporting of side effects

If you get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in By reporting side effects you can help provide more information on the safety of this medicine.

5. How to store Orphacol

Keep out of the reach and sight of children.

Do not use Orphacol after the expiry date which is stated on the carton and blister after EXP. The expiry date refers to the last day of that month.

Store below 30°C.

Medicines should not be disposed of via wasterwater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.

6. Contents of the pack and other information

What Orphacol contains

• – The active substance is cholic acid.

Orphacol 50 mg : Each hard capsule contains 50 mg of cholic acid.

Orphacol 250 mg : Each hard capsule contains 250 mg of cholic acid.

• – The other ingredients are:

What Orphacol looks like and contents of the pack

Orphacol is available as hard capsules (capsules) of oblong shape. Capsules of 50 mg cholic acid are blue and white and capsules of 250 mg cholic acid are green and white. They are contained in blisters of 10 capsules each.

Pack sizes are available in 30, 60 and 120 capsules.

Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder

Laboratoires CTRS

63, rue de l’Est

92100 Boulogne-Billancourt

France

Manufacturer

Laboratoires CTRS

63, rue de l'Est

92100 Boulogne-Billancourt

France

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

België/Belgiqu­e/Belgien Laboratoires CTRS Tél/Tel: +32 (0)2 40 11 442	Lietuva Immedica Pharma AB Tel/Puh: +46 (0)8 533 39 500
Efc^rapufl Laboratoires CTRS Ten.: + 33 (0)1 707 60 637	Luxembourg/Lu­xemburg Laboratoires CTRS Tél/Tel: +352 278 62 329
Česká republika Laboratoires CTRS Tel.: + 33 (0)1 70 76 06 37	Magyarország Medis Hungary Kft Tel: +36 (2) 380 1028

Danmark Immedica Pharma AB Tlf: +46 (0)8 533 39 500	Malta Laboratoires CTRS Tel: +356 2776 1358
Deutschland Laboratoires CTRS Tel: +49 (0)3022153008	Nederland Laboratoires CTRS Tel: +31 (0)2 070 38 155
Eesti Immedica Pharma AB Tel/Puh: +46 (0)8 533 39 500	Norge Immedica Pharma AB Tel/Puh: +46 (0)8 533 39 500
EXÀàôa Laboratoires CTRS Tel: + 33 (0)1 70 76 06 37	Österreich Laboratoires CTRS Tel: +43 (0)7 208 16 847
España Laboratoires CTRS Tel: + (34) 914 146 613	Polska Laboratoires CTRS Tel.: + 33 (0)1 70 76 06 37
France Laboratoires CTRS Tél: + 33 (0)1 70 76 06 37	Portugal Laboratoires CTRS Tel: + 33 (0)1 70 76 06 37
Hrvatska Medis Adria d.o.o. Tel: +385 (1) 230 3446	România Laboratoires CTRS Tel: + 33 (0)1 70 76 06 37
Ireland Laboratoires CTRS Tel: +353 (0)1 695 00 63	Slovenija Medis d.o.o. Tel: +386 (1) 589 6900
Ísland Immedica Pharma AB Sími: + 46 (0)8 533 39 500	Slovenská republika Laboratoires CTRS Tel: + 33 (0)1 70 76 06 37
Italia Laboratoires CTRS Tel: +39 (0)687 501302	Suomi/Finland Immedica Pharma AB Tel/Puh: +46 (0)8 533 39 500

Kûnpoç

Laboratoires CTRS

Tel: + 33 (0)1 70 76 06 37

Latvija

Immedica Pharma AB

Tel: +46 (0)8 533 39 500

This leaflet was last revised in

This medicine has been authorised under “Exceptional Circumstances”.

This means that because of the rarity of this disease and for ethical reasons it has been impossible to get complete information on this medicine.

The European Medicines Agency will review any new information on the medicine every year and this leaflet will be updated as necessary.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site:. There are also links to other websites about rare diseases and treatments.

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