Orphacol 50 mg hard capsules

Package leaflet: Information for the user

Introduction

PACKAGE LEAFLET: INFORMATION FOR THE USER

Orphacol 50 mg hard capsules

Orphacol 250 mg hard capsules Cholic acid

This medicinal product is subject to additional monitoring, which will allow for faster detection of new safety information. You can help by reporting any adverse reactions you may experience. Section 4, towards the end, includes information on how to report these adverse reactions.

• Read the entire leaflet carefully before you start taking this medicine.

• Keep this leaflet, as you may need to read it again.

• If you have any questions, ask your doctor or pharmacist.

• This medicine has been prescribed for you and must not be given to other people, even if they have the same symptoms, as it may harm them.

• If you experience any adverse reactions, consult your doctor or pharmacist, even if they are

adverse reactions not listed in this leaflet. See section 4.

Leaflet contents:

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 adverse reactions
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 medical disorders are caused by defects in bile acid production, and Orphacol is used for the treatment of infants from 1 month of age up to 2 years of age, children, adolescents, and adults with these disorders. The cholic acid contained in Orphacol replaces the bile acids that are lacking due to the defect in bile acid production.

2. What you need to know before starting to take 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, a medicine used to treat epilepsy.

Warnings and precautions

Your doctor will arrange for you to have blood and urine tests and liver function tests when you start treatment and periodically thereafter. Do not forget to visit your doctor for these regular check-ups at least once a year.

Use of Orphacol with other medicines

Inform 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 take these medicines, you must take Orphacol at least 5 hours before or at least 5 hours after taking those 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 these medicines.

Pregnancy and breastfeeding

Consult your doctor if you are planning to become pregnant. Take a pregnancy test as soon as you suspect you might be pregnant. It is very important that you continue taking Orphacol during pregnancy.

If you become pregnant while taking Orphacol, your doctor will decide on the treatment and dosage most suitable for your condition. As a precautionary measure, your doctor will closely monitor your health and that of the fetus throughout the pregnancy.

Orphacol may be administered during breastfeeding. Inform your doctor if you plan to start or have already started breastfeeding before taking Orphacol.

Consult your doctor or pharmacist before taking any medicine.

Driving and using machines

Orphacol is not expected to affect the ability to drive or use machines.

Orphacol contains lactose

Orphacol contains a sugar (monohydrate lactose). If your doctor has informed you that you have an intolerance to certain sugars, consult him/her before taking this medicine.

3. How to take Orphacol

Follow exactly the instructions for administering Orphacol as given by your doctor. If in doubt, consult your doctor.

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

Before treatment begins, your doctor will evaluate the results of your laboratory tests to determine the correct dose for your case. Your doctor will subsequently adjust the dose depending on your body's response.

Orphacol capsules must be taken with food, approximately at the same time each day, in the morning and/or evening. Taking Orphacol at the same time each day with a meal will help you remember to take this medicine and may also help your body absorb it better. The capsules should be swallowed whole with water, without chewing.

If your doctor has prescribed a dose requiring you to take more than one capsule per day, you and your doctor will decide how to divide the doses throughout the day. For example, you may take one capsule in the morning and another in the evening. This way, you will need to take fewer capsules at one time.

Use in children

In infants and children who cannot swallow capsules, the capsules may be opened and their contents mixed with an infant formula or a young child-appropriate apple/orange or apple/apricot juice.

If you take more Orphacol than you should

If you take more Orphacol than you should, contact your doctor as soon as possible. Your doctor will evaluate your laboratory test results and advise you whether to resume your treatment with the normal dose.

If you forget to take Orphacol

Take the next dose at your usual scheduled time. Do not take a double dose to make up for forgotten doses.

If you stop taking Orphacol

If you stop treatment with Orphacol, you risk suffering irreversible liver damage. Never stop treatment with Orphacol unless instructed by your doctor.

If you have any further questions about the use of this product, ask your doctor.

4. Possible adverse effects

Like all medicines, Orphacol may produce adverse effects, although not everyone experiences them.

Some patients have experienced itching and/or diarrhoea, but the likelihood of these occurring is unknown (frequency cannot be estimated from the available data). If you experience itching or diarrhoea for more than three days, inform your doctor.

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

Reporting of adverse effects

If you experience any type of adverse effect, consult your doctor, even if it is a possible adverse effect not listed in this leaflet. You may also report them directly through the national reporting system included in Annex V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Orphacol

Keep out of the reach and sight of children.

Do not use Orphacol after the expiry date stated on the cardboard box and blister after "EXP". The expiry date refers to the last day of the month indicated.

Store below 30°C.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and packaging that you no longer need. This will help protect the environment.

6. Package contents and other information

Composition of Orphacol

• 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 components are:

Capsule contents: Monohydrate lactose, colloidal anhydrous 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);

Appearance of the product and package contents

Orphacol is available as oblong-shaped hard capsules. The 50 mg cholic acid capsules are blue and white, and the 250 mg capsules are green and white. The capsules are supplied in blisters of 10 capsules.

The medicine is marketed in pack sizes of 30, 60 and 120 capsules. Some pack sizes may not be available.

Marketing Authorization Holder and Manufacturer

Marketing Authorization Holder

THERAVIA

16 Rue Montrosier

92200 Neuilly-sur-Seine

France

Manufacturer

THERAVIA

16 Rue Montrosier

92200 Neuilly-sur-Seine

France

For more information about this medicine, please contact the local representative of the Marketing Authorization Holder:

Date of the most recent review of this leaflet:

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 medicine.

The European Medicines Agency will review any new information about the medicine annually, and this leaflet will be updated as necessary.

Other sources of information

Detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu. The site also provides links to other websites on rare diseases and orphan medicinal products.