What is Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid used for?
Chenodeoxycholic acid sigma-tau is a medicine that contains the active substance chenodeoxycholic acid. It is a "primary bile acid", which is one of the main components of bile (a fluid produced by the liver that helps the digestion of fats).
Chenodeoxycholic acid sigma-tau is used in the treatment of adults and children from one month of age with cerebrotendinous xanthomatosis. These patients are unable to produce the primary bile acid chenodeoxycholic acid in sufficient quantities due to genetic abnormalities that cause a lack of the hepatic enzyme sterol 27-hydroxylase. When primary bile acids are lacking, the body instead produces abnormal bile acids and other substances, which accumulate throughout the body causing damage.
Because the number of patients with this condition is low, the disease is considered 'rare' and Chenodeoxycholic acid sigma-tau was designated an 'orphan medicine' (a medicine used in rare diseases) on 16 December 2014. Chenodeoxycholic acid sigma-tau is a "hybrid medicine".
This means that it is similar to a "reference medicine" that contains the same active ingredient. The reference medicine for Chenodeoxycholic acid sigma-tau is Xenbilox. However, Xenbilox differs from Chenodeoxycholic acid sigma-tau in that it is authorized for a different use (to dissolve cholesterol gallstones).
How is Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid used?
Chenodeoxycholic acid sigma-tau can only be obtained with a prescription. Treatment should be initiated and supervised by a physician experienced in the treatment of cerebrotendinous xanthomatosis or other diseases concerning the production of primary bile acids.
Chenodeoxycholic acid sigma-tau is available as capsules (250 mg) to be taken 3 times a day at about the same time each day. The daily dose is determined and adjusted during treatment for each patient according to age, liver function and bile acid levels in the blood and urine. For small children and children who are unable to swallow the capsules, the contents of the latter can be mixed with 8.4% sodium bicarbonate solution to form a liquid.
For more information, see the package leaflet.
How does Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid work?
Chenodeoxycholic acid is one of the main primary bile acids produced by the liver. The chenodeoxycholic acid contained in this medicine replaces the one missing by the patient. This helps reduce the production of abnormal substances and contributes to the normal activity of bile in the digestive system, thereby alleviating the symptoms of the disease.
What benefit has Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid shown during my studies?
Chenodeoxycholic acid sigma-tau was analyzed in a study that examined the records of 35 patients with cerebrotendinea xanthomatosis treated with chenodeoxycholic acid for about 9 years. Out of 23 patients for whom bile acid levels were available, all had a reduction in their levels (averaging 56-69 µmol / l). Out of 14 patients for whom bile acid levels were available in urine, 79% (11 of 14) had reduced levels in the most recent analysis. Most of the patients also showed improvements in the symptoms of the disease: all reported an improvement in diarrhea, 89% of the patients showed an improvement in mental faculties, 60% of the patients showed an improvement in mobility, while 85% showed 77% of patients showed psychiatric improvement based on two different scores.
What are the risks associated with Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid?
The side effects seen with Chenodeoxycholic acid sigma-tau are constipation and abnormal liver test values; however, from the available data their frequency could not be reliably estimated. They were mild to moderate in severity and not durable.
For the list of limitations, see the package leaflet.
Why has Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid been approved?
Chenodeoxycholic acid has been used in the treatment of cerebrotendinous xanthomatosis for about 40 years, although it was not authorized for such use. However, given the rarity of the disease, the available data on the use of the medicine are still limited. Nevertheless, studies have shown that the medicine has beneficial effects on patients and has no significant side effects. Therefore, the Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Chenodeoxycholic acid sigma-tau's benefits are greater than its risks and recommended that it be approved for use in the EU.
Chenodeoxycholic acid sigma-tau was authorized in "exceptional circumstances" because it was not possible to obtain complete information on it 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 Chenodeoxycholic acid sigmatau?
Since Chenodeoxycholic acid sigma-tau was authorized in exceptional circumstances, the company that markets it will set up a register to monitor its benefits and safety.
What measures are being taken to ensure the safe and effective use of Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic Acid?
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Chenodeoxycholic acid sigma-tau have been included in the summary of product characteristics and the package leaflet.
Further information on Chenodeoxycholic Acid Sigma-Tau - Chenodeoxycholic acid
For the full EPAR version of Chenodeoxycholic acid sigma-tau, see the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about treatment with Chenodeoxycholic acid sigma-tau, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products related to Chenodeoxycholic acid sigma-tau is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
