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Overview
This is a summary of the European public assessment report (EPAR) for Orphacol. It explains how the European Medicines Agency assessed the medicine to recommend its authorisation in the European Union (EU) and its conditions of use. It is not intended to provide practical advice on how to use Orphacol.
For practical information about using Orphacol, patients should read the package leaflet or contact their doctor or pharmacist.
Orphacol is a medicine containing cholic acid, a substance found in the bile, which is used to digest fats.
It is used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. Orphacol is used in patients who do not have enough of two specific liver enzymes (3?-hydroxy-?5-C27-steroid oxidoreductase or ?4-3-oxosteroid-5?-reductase). This makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure.
Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Orphacol was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 December 2002.
The medicine can only be obtained with a prescription and treatment with Orphacol should be started and supervised by a doctor specialised in liver disease.
Orphacol is available as capsules and should be taken every day at approximately the same time, with a meal. The daily dose is between 5 and 15 mg per kilogram body weight, adjusted for each patient according to the state of their bile acid, with a minimum daily dose of 50 mg and a maximum of 500 mg. For small children who cannot swallow capsules, the contents can be mixed in with infant formula or juice. Treatment should be stopped if liver function does not improve within three months.
Cholic acid is the main primary bile acid produced by the liver. The cholic acid contained in Orphacol replaces the patient’s missing cholic acid. This helps to decrease the production of abnormal bile acids and contributes to the normal activity of bile in the digestive system, thereby relieving the symptoms of the condition.
Because cholic acid is a well known substance, and its use in these enzyme deficiencies is well established, the applicant presented data from the scientific literature. The applicant presented data on 49 patients with inborn errors in primary bile acid synthesis, taken from the scientific literature (38 patients with 3?-hydroxy-?5-C27-steroid oxidoreductase deficiency and 11 patients with ?4-3-oxosteroid-5?-reductase deficiency). It compared the outcomes of 28 patients who received cholic acid with others who were given different bile acids or did not receive bile-acid treatment.
In the scientific literature, cholic acid treatment was shown to reduce the amount of abnormal bile acids in patients, to restore the normal functions of the liver and to help delay or prevent the need for a liver transplant.
The side effects seen with Orphacol were diarrhoea, pruritus (itching), increases in transaminases (liver enzymes) and possibly gallstones, although their frequency could not be reliably estimated from the available limited data.
Orphacol should not be used in people who may be hypersensitive (allergic) to cholic acid or any of the other ingredients. It must not be taken by patients already taking phenobarbital, a medicine used to treat epilepsy.
The Committee for Medicinal Products for Human Use (CHMP) noted that the use of cholic acid to treat inborn errors in primary bile-acid synthesis was well established in medical practice and documented in the scientific literature, although the number of documented cases was low due to the rarity of the condition. It decided that based on the evidence from the scientific literature, Orphacol’s benefits are greater than its risks and recommended that it be given marketing authorisation.
Orphacol has been authorised under ‘exceptional circumstances’. This means that because the disease is rare, it has not been possible to obtain complete information about Orphacol. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.
Since Orphacol has been approved under exceptional circumstances, the company that markets Orphacol will establish a database of patients treated with Orphacol to monitor the safety and effectiveness of the treatment, and will submit the results to the CHMP at regular, specified intervals.
A risk-management plan has been developed to ensure that Orphacol is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Orphacol, including the appropriate precautions to be followed by healthcare professionals and patients.
The company that makes Orphacol will also provide doctors in all Member States who will use Orphacol with a pack containing product literature and information regarding the correct diagnosis of these disease conditions, the risks of side effects and how to use the medicine properly.
The European Commission granted a marketing authorisation valid throughout the European Union for Orphacol on 12 September 2013.
For more information about treatment with Orphacol, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
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Latest procedure affecting product information:
PSUSA-10208-202409
10/04/2025
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
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Product details
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Name of medicine
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Orphacol
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Active substance
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cholic acid
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International non-proprietary name (INN) or common name
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cholic acid
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Therapeutic area (MeSH)
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- Digestive System Diseases
- Metabolism, Inborn Errors
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Anatomical therapeutic chemical (ATC) code
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A05AA03
Pharmacotherapeutic group
Bile acids and derivatives
Therapeutic indication
Orphacol is indicated for the treatment of inborn errors in primary bile-acid synthesis due to 3?-hydroxy-?5-C27-steroid oxidoreductase deficiency or ?4-3-oxosteroid-5?-reductase deficiency in infants, children and adolescents aged one month to 18 years and adults.
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