What is Orphacol - Cholic acid used for and what is it used for?
Orphacol is a medicine containing cholic acid, a substance present in bile that is used to digest fats. 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 possess a sufficient quantity of two specific liver enzymes (3β-hydroxy-Δ5-C27-steroid oxidoreductase or 3-oxo-Δ4-steroid-5β-reductase). This deficit compromises the ability of the liver to produce a sufficient quantity of the main components of bile, the so-called primary bile acids, such as cholic acid. When there is an insufficiency of these primary bile acids, the body produces abnormal bile acids that can damage the liver, generating 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 as an "orphan medicine" (a medicine used in rare diseases) on December 18, 2002.
How is Orphacol - Cholic acid used?
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 as a capsule and should be taken at about the same time each day during a meal. The daily dose is between 5 and 15 mg per kg of body weight, adequate for each patient based on the state of bile acid, starting from a minimum daily dose of 50 mg up to a maximum dose of 500 mg. In children who cannot swallow the capsules, their contents can be added to the milk of the bottle or to a fruit juice. Treatment should be stopped if the liver function does not improve over three months.
How does Orphacol - Cholic acid work?
Cholic acid is the main primary bile acid produced by the liver. Cholic acid in Orphacol replaces the cholic acid that should be produced by the patient's body. In this way it helps to reduce the production of abnormal bile acids and promotes the normal activity of bile in the digestive system, alleviating 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 enzymatic deficits is consolidated, the applicant presented data from the scientific literature. The applicant presented data from the scientific literature, collected on 49 patients with congenital errors of primary bile acid synthesis (38 patients with 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency and 11 patients with 3-oxo-Δ4 deficiency -steroide-5β-reductase). 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 subjected to 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 helps to delay or prevent the need for a liver transplant.
What is the risk associated with Orphacol - Cholic acid?
The side effects seen with Orphacol are diarrhea, itching, increased transaminases (liver enzymes) and sometimes gallstones, although their frequency cannot be reliably estimated based on the limited data available. Orphacol should not be used in people who may be hypersensitive (allergic) to cholic acid or any of the other ingredients. It should not be given to 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 consolidated in medical practice and well documented in the scientific literature, although the number of documented cases are 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 marketing authorization. Orphacol was authorized in "exceptional circumstances" because complete information on Orphacol could not be obtained due to the rarity of the disease. Every 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?
Because Orphacol has been authorized in 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 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 intend to prescribe Orphacol with a dossier containing product literature and information regarding the correct diagnosis of these conditions, the risks of side effects and the correct use of the medicine.
More information on Orphacol - Cholic acid
On 12 September 2013, the European Commission issued a marketing authorization for Orphacol, valid throughout the European Union. For more information about treatment with Orphacol, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. The summary of the opinion of the Orphacol Committee for Orphan Medicinal Products is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation. Last update of this summary: 09-2013.
