PATIENT INFORMATION LEAFLET

Orphacol 50 mg Hard Capsules

Orphacol 250 mg Hard Capsules Magnesium Hydroxide

This medicine is subject to additional monitoring. This will help speed up the detection of any new information about its safety. You can contribute by reporting any side effects you may get. The final part of section 4 includes information on how to report these side effects.

• Read the entire leaflet carefully before starting to take the medicine.

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

- If you have any doubts, consult your doctor or pharmacist.

- This medicine has been prescribed for you and should not be given to others, even if they have the same symptoms, as it may harm them.

- If you experience side effects, consult your doctor or pharmacist, even if they are not listed in this leaflet. See section 4.

1. What is Orphacol and what it is used for

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

3. How to take Orphacol

4. Possible side effects

5. Storage of Orphacol

6. Contents of the pack and additional information

Orphacol contains glycocholic acid, a bile acid normally produced by the liver. Certain medical conditions are caused by defects in the production of bile acids, and Orphacol is used for the treatment of infants from 1 month of age to 2 years of age, children, adolescents, and adults with those conditions. The glycocholic acid contained in Orphacol replaces the missing bile acids due to the defect in bile acid production.

Do not take Orphacol

- if you are allergic to phenolphthalein or to any of the other ingredients of this medicine (listed in section 6).

- if you are taking phenobarbital, a medicine used for the treatment of epilepsy.

Warnings and precautions

Your doctor will have you undergo blood and urine tests and liver function tests when you start treatment and at regular intervals thereafter. Do not forget to visit your doctor for these periodic examinations at least once a year.

Use of Orphacol with other medicines

Inform your doctor or pharmacist if you are using, have used recently, or may have to use any other medicine.

Some medicines used to lower cholesterol levels, known as bile acid sequestrants (colestiramine, colestipol, colesevelam), and medicines used to treat heartburn that contain aluminum, may reduce the effect of Orphacol. If you take those medicines, you should take Orphacol at least 5 hours before or at least 5 hours after those other medicines. Cyclosporin (a medicine used for immunosuppression) may also alter the effect of Orphacol. Inform your doctor if you are using those medicines.

Pregnancy and breastfeeding

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

If you become pregnant during treatment with Orphacol, your doctor will decide on the best treatment and dosage for your situation. As a precaution, your doctor will closely monitor your condition and that of the fetus throughout the pregnancy.

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

Consult your doctor or pharmacist before taking any medicine.

Driving and operating machinery

Orphacol is not expected to affect your ability to drive and operate machinery.

Orphacol contains lactose

Orphacol contains a sugar (lactose monohydrate). If your doctor has told you that you have an intolerance to some sugars, consult them before taking this medicine.

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

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

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

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

If your doctor has prescribed a dose that requires you to take more than one capsule per day, you and your doctor will decide how to take it during the day. For example, you can take one capsule in the morning and another in the evening. This way, you will have to take fewer capsules at one time.

Use in children

In infants and children who cannot swallow capsules, the capsules can be opened and their contents added to an infant formula or a juice adapted for young children, such as apple or orange juice.

If you take more Orphacol than you should

If you take more Orphacol than you should, contact your doctor as soon as possible. The doctor will evaluate your test results and indicate if you should resume your treatment with the normal dose.

If you forget to take Orphacol

Take the next dose at the time it is normally due. Do not take a double dose to compensate for the missed doses.

If you interrupt treatment with Orphacol

If you interrupt treatment with Orphacol, you risk suffering irreversible liver damage. Never interrupt treatment with Orphacol unless your doctor tells you to.

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

Like all medications, Orphacol may produce adverse effects, although not all people will experience them.

Some patients have experienced itching and/or diarrhea, but the probability of this occurring cannot be estimated from the available data. If you experience itching or diarrhea for more than three days, inform your doctor.

Biliary calculi have been reported after long-term treatment with Orphacol.

Reporting 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 prospectus. You can also report them directly through the national notification system included in theAnnex V.By reporting adverse effects, you can contribute to providing more information about the safety of this medication.

Keep out of reach and sight of children.

Do not use Orphacol after the expiration date that appears on the carton box and the blister after "CAD". The expiration date is the last day of the month indicated.

Store below 30°C.

Medicines should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the packaging and medicines you no longer need. In this way, you will help protect the environment.

Composition of Orphacol

- The active ingredient is glycolic acid.

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

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

- The other components are:

Hard capsule content: Lactose monohydrate, anhydrous colloidal silica, magnesium stearate

Capsule coating:

Orphacol 50 mg: gelatin, titanium dioxide (E171), indigo carmine (E132);

Orphacol 250 mg: gelatin, titanium dioxide (E171), indigo carmine (E132), iron oxide yellow (E172);

Appearance of the product and contents of the pack

Orphacol is presented in hard, oblong capsules. The 50 mg glycolic acid capsules are blue and white, and the 250 mg capsules are green and white. They are presented in blisters of 10 capsules.

The medicinal product is marketed in pack sizes of 30, 60 and 120 capsules. Only some pack sizes may be marketed.

Holder of the marketing authorisation and responsible person for the manufacture

Holder of the marketing authorisation

THERAVIA

16 Rue Montrosier

92200 Neuilly-sur-Seine

France

Responsible person for the manufacture

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:

Last update of the summary of product characteristics:

This medicinal product has been authorised under «Exceptional Circumstances»,

This authorisation route means that due to the rarity of the disease and for ethical reasons it has not been possible to obtain a complete overview of this medicinal product.

The European Medicines Agency will review annually any new information about the medicinal product that may become available and this summary of product characteristics will be updated as necessary.

Other sources of information

Further information on this medicinal product is available on the website of the European Medicines Agencyhttp://www.ema.europa.eu.Tambiénprovides links to other websites on rare diseases and orphan medicinal products.