Orphacol - Cholic acid

What is Orphacol - Cholic acid and what is it used for?

Orphacol is a medicine containing cholic acid, a substance found in bile that is used to digest fat. Orphacol is indicated for the treatment of adults and children from one month of age who are unable to produce bile due to a "genetic abnormality. It is used in patients who do not have sufficient amounts of two specific liver enzymes ( 3β-hydroxy-Δ5-C27-steroid oxidoreductase or 3-oxo-Δ4-steroid-5β-reductase). "cholic acid. When there is an insufficiency of these primary bile acids, the body produces abnormal bile acids which can damage the liver, resulting in severe liver failure. Since the number of patients with inborn errors of primary bile acid synthesis is low, the disease is considered 'rare', and Orphacol was designated an 'orphan medicine' (a medicine used in rare diseases) on 18 December 2002.

How is Orphacol used - Cholic acid?

The medicine can only be obtained with a prescription and treatment with Orphacol should be started and monitored by a doctor who specializes in liver disease. Orphacol is available in capsule form and should be taken at approximately the same time each day with a meal. The daily dose is between 5 and 15 mg per kg of body weight, adjusted for each patient according to the bile acid status, starting from a minimum daily dose of 50 mg up to a maximum dose of 500 mg. In children who cannot swallow capsules, their contents can be added to bottle milk or fruit juice. Treatment should be stopped if liver function does not improve within three months.

How does Orphacol - Cholic acid work?

Cholic acid is the main primary bile acid produced by the liver. Cholic acid contained in Orphacol replaces the cholic acid that should be produced by the patient's body. Doing so helps reduce the production of abnormal bile acids and promotes the normal activity of bile in the digestive system, relieving the symptoms of the condition.

What benefit has Orphacol - Cholic acid shown during the studies?

Since cholic acid is a known substance and its use in these enzyme deficiencies is well established, the applicant presented data from the scientific literature. The applicant presented data from the scientific literature, collected on 49 patients with inborn errors of synthesis of the primary bile acids (38 patients with 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency and 11 patients with 3-oxo-Δ4-steroid-5β-reductase deficiency). He then compared the effects on 28 subjects treated with cholic acid compared to the effects on patients treated with different bile acids or not undergoing any substitution therapy. From the scientific literature it emerged that treatment with cholic acid reduces the amount of abnormal bile acids in patients, restores normal liver functions and contributes to delaying or prevent the need for a liver transplant.

What is the risk associated with Orphacol - Cholic acid?

Side effects seen with Orphacol are diarrhea, itching, increased transaminases (liver enzymes) and sometimes gallstones, although their frequency cannot be reliably estimated from the limited data available. Orphacol must not be used in people who may be hypersensitive (allergic) to cholic acid or any of the other ingredients. It must not be used in patients who are taking phenobarbital, a medicine used to treat epilepsy.

Why has Orphacol - Cholic acid been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) noted that the use of cholic acid in the treatment of inborn errors of primary bile acid synthesis is well established in medical practice and well documented in the scientific literature, although the number of documented cases is low due to the rarity of the condition. He therefore decided that Orphacol's benefits are greater than its risks and recommended that it be given a 'marketing authorization' for the medicine. Orphacol was authorized under 'exceptional circumstances' because it was not possible to obtain complete information on Orphacol due to the rarity of the disease. Each year the European Medicines Agency will review the new information available and this summary will be updated accordingly.

What information is still awaited for Orphacol - Cholic acid?

As Orphacol has been authorized under exceptional circumstances, the company that markets Orphacol will create a database of patients treated with Orphacol to monitor the safety and efficacy of the treatment and will present the results to the CHMP at regular and specific intervals.

What measures are being taken to ensure the safe and effective use of Orphacol - Cholic acid?

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 added to the summary of product characteristics and 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 wishing to prescribe Orphacol with a dossier containing literature on the product and information relating to the correct diagnosis of these conditions, the risks of side effects and the correct use of the medicine.

More information about Orphacol - Cholic acid

On 12 September 2013, the European Commission granted a "Marketing Authorization" for Orphacol, valid throughout the European Union. For more information on Orphacol therapy, read the package leaflet (included with the EPAR) or consult your doctor. or the pharmacist. The summary of the opinion of the Committee for Orphan Medicinal Products for Orphacol is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation. Last update of this summary: 09-2013.


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