Orphacol

Package leaflet: Information for the user

Orphacol 50 mg hard capsules, 250 mg hard capsules

Cholic acid
This medicinal product is subject to additional monitoring. This will allow for the rapid identification of new safety information. You can help by reporting any side effects you experience while taking this medicinal product. See the end of section 4 for information on how to report side effects.
Please read all of this leaflet carefully before you start 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 their symptoms are the same as yours, as it may be harmful.
• If you experience any side effect, including those not listed in this leaflet, tell your doctor or pharmacist. See section 4.

Contents of this leaflet

1. What Orphacol is and what it is used for
2. What you need to know before taking 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 normally produced by the liver. Certain diseases are caused by defects in bile acid production, and Orphacol is used to treat infants from 1 month to 2 years of age, children, adolescents, and adults with these diseases. The cholic acid contained in Orphacol replaces the bile acids missing due to the defect in bile acid production.

2. What you need to know before taking Orphacol

Do not take Orphacol

• if you are allergic to cholic acid or to any of the other ingredients of this medicine (listed in section 6).
• if you are taking phenobarbital or primidone, a medicine used to treat epilepsy.

Warnings and precautions
During treatment, your doctor will carry out various blood and urine tests at different times to monitor
how your body is responding to the medicine and to help determine the dose you need. If you are growing rapidly or become ill (e.g. if you have liver problems), more frequent testing will be required.

Other medicines and Orphacol
Tell your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines.
Some medicines used to lower cholesterol levels, known as bile acid sequestrants (cholestyramine,
colestipol, colesevelam), and medicines used to treat heartburn containing aluminium,
may reduce the effect of Orphacol. If you are taking these medicines, take Orphacol at least 5 hours
before or at least 5 hours after taking the other medicines. Cyclosporine (a medicine used
to suppress the immune system) may also alter the effect of Orphacol. Inform your doctor if you are
taking cyclosporine.
Ursodeoxycholic acid may reduce the effect of Orphacol if both medicines are taken at the same time. If you are prescribed ursodeoxycholic acid together with Orphacol as single daily doses, take one medicine in the morning and the other in the evening. If you are prescribed separate doses of ursodeoxycholic acid and/or Orphacol, ask your doctor or pharmacist for advice on the correct sequence of administration, as these medicines must be taken several hours apart.

Pregnancy and breastfeeding
Ask your doctor for advice if you are planning a pregnancy. Perform a pregnancy test as soon as you suspect you might be pregnant. It is very important to continue taking Orphacol during pregnancy.
If you become pregnant while being treated with Orphacol, your doctor will decide which treatment and dosage are best suited to your situation. As a precaution, you and your baby should be closely monitored during pregnancy.
Orphacol may be used during breastfeeding. Before starting Orphacol, inform your doctor if you plan to breastfeed or are already breastfeeding.
Ask your doctor or pharmacist for advice before taking any medicine.

Driving and using machines
Orphacol is unlikely to affect your ability to drive or use machinery.

Orphacol contains lactose
Orphacol contains a sugar (monohydrate lactose). If your doctor has informed you that you are intolerant to certain sugars, consult your doctor before taking Orphacol.

3. How to take Orphacol

Take this medicine exactly as your doctor has told you. If you have any doubts, consult your doctor.

The usual dose is 5 to 15 mg per kg of body weight daily in infants, children, adolescents, and adults.
Before starting treatment, your doctor will review laboratory test results to determine the correct dose for you. The dose may then be further adjusted by your doctor depending on your body's response.

Orphacol capsules must be taken orally with food, approximately at the same time each day, in the morning and/or evening. Taking Orphacol regularly with meals will help you remember to take it and may also help your body absorb it better. The capsules must be swallowed whole with water. Do not chew them.

If your doctor has prescribed a dose requiring more than one capsule per day, you and your doctor may decide how to divide this dose throughout the day. For example, you may take one capsule in the morning and one in the evening. This way, you will take fewer capsules at each time. However, this may not be possible if you are also prescribed another medicine containing ursodeoxycholic acid. In this case, you must ask your doctor or pharmacist for advice on the correct sequence of administration, as ursodeoxycholic acid and Orphacol must be taken several hours apart (see section 2).

Use in children
For newborns and children who cannot swallow capsules, the capsules may be opened and their contents mixed with infant milk or apple/orange or apple/apricot juice for infants.

If you take more Orphacol than you should
If you have taken more Orphacol than you should, contact your doctor as soon as possible. Your doctor will evaluate laboratory tests and advise you on when to resume treatment at the normal dose.

If you forget to take Orphacol
Take the next dose at the usual time. Do not take a double dose to make up for the forgotten dose.

If you stop taking Orphacol
If you stop treatment with Orphacol, there is a risk of causing irreversible liver damage. You must never stop taking Orphacol unless your doctor advises you to do so.

If you have any questions about 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.
Some 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). Inform your doctor if itching and/or diarrhoea lasts longer than three days.
In various patients, an increase in liver enzymes (serum transaminases) has been reported during treatment with Orphacol (frequency cannot be determined from the available data). Your doctor will decide what action to take should this occur.
Gallstones have been reported after long-term therapy with Orphacol.

Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, talk to your doctor. You can also report side effects directly via the national reporting system listed in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store Orphacol

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 and on the blister after "Exp.".
The expiry date refers to the last day of that month.
Store below 25 °C.
Do not dispose of any medicine via wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Package contents 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 excipients are: Capsule contents: lactose monohydrate (for further information see section 2 under “Orphacol contains lactose”), anhydrous colloidal silica, magnesium stearate. Capsule shell: Orphacol 50 mg: gelatin, titanium dioxide (E171), indigo carmine (E132); Orphacol 250 mg: gelatin, titanium dioxide (E171), indigo carmine (E132), yellow iron oxide (E172).

Description of the appearance of Orphacol and the contents of the pack
Orphacol is available as oblong-shaped hard capsules. The 50 mg cholic acid capsules are blue and white, and the 250 mg cholic acid capsules are green and white. They are supplied in blisters of 10 capsules each.
Pack sizes of 30, 60 and 120 capsules are available.
Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Marketing Authorisation Holder
THERAVIA
16 Rue Montrosier
92200 Neuilly-sur-Seine
France

Manufacturer
THERAVIA
16 Rue Montrosier
92200 Neuilly-sur-Seine
France

For further information about this medicinal product, please contact the local representative of the Marketing Authorisation Holder:

België/Belgique/Belgien Lietuva
THERAVIA Immedica Pharma AB
Tel/Puh: +46 (0)8 533 39 500
Tél/Tel: +32 (0)2 808 2973 [email protected]
[email protected]

България Luxembourg/Luxemburg
THERAVIA THERAVIA
Teл.: +33 (0)1 72 69 01 86 Tél/Tel: +352 278 62 329
[email protected] [email protected]

Česká republika Magyarország
THERAVIA Medis Hungary Kft
Tel.: +33 (0)1 72 69 01 86 Tel: +36 (2) 380 1028
[email protected] [email protected]

Danmark
Immedica Pharma AB
Tlf: +46 (0)8 533 39 500
[email protected]

Deutschland Nederland
THERAVIA THERAVIA
Tel: 08001090001 Tel : +31 (0)20 208 2161
[email protected] [email protected]

Eesti Norge
Immedica Pharma AB Immedica Pharma AB
Tel/Puh: +46 (0)8 533 39 500 Tel/Puh: +46 (0)8 533 39 500
[email protected] [email protected]

Ελλάδα
THERAVIA
Τηλ: +33 (0)1 72 69 01 86
[email protected]

España Polska
THERAVIA THERAVIA
Tel: + (34) 914 146 613 Tel.: +33 (0)1 72 69 01 86
[email protected] [email protected]

France Portugal
THERAVIA THERAVIA
Tél: +33 (0)1 72 69 01 86 Tel: 800210571
[email protected] [email protected]

Hrvatska România
Medis Adria d.o.o. THERAVIA
Tel: +385 (1) 230 3446 Tel: +33 (0)1 72 69 01 86
[email protected] [email protected]

Ireland Slovenija
THERAVIA Medis d.o.o.
Tel : +353-(0)1-903 8043 Tel: +386 (1) 589 6900
[email protected] [email protected]

Ísland Slovenská republika
Immedica Pharma AB THERAVIA
Sími: + 46 (0)8 533 39 500 Tel: +33 (0)1 72 69 01 86
[email protected] [email protected]

Italia Suomi/Finland
THERAVIA Immedica Pharma AB
Tel : 800 959 161 Tel/Puh: +46 (0)8 533 39 500
[email protected] [email protected]

Κύπρος Sverige
THERAVIA Immedica Pharma AB
Τηλ: +33 (0)1 72 69 01 86 Tel: +46 (0)8 533 39 500
[email protected] [email protected]

Latvija
Immedica Pharma AB
Tel: +46 (0)8 533 39 500
[email protected]

This medicinal product has been authorised under "exceptional circumstances". This means that, due to the rarity of the disease and for ethical reasons, it has not been possible to obtain complete information about this medicinal product.
The European Medicines Agency will review any new information on this medicinal product annually, and this patient information leaflet will be updated if necessary.

Other sources of information
More detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu/. In addition, links to other websites on rare diseases and their treatments are provided.