Overview

This is a summary of the European public assessment report (EPAR) for Chenodeoxycholic acid Leadiant. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Chenodeoxycholic acid Leadiant.

For practical information about using Chenodeoxycholic acid Leadiant, patients should read the package leaflet or contact their doctor or pharmacist.

Chenodeoxycholic acid Leadiant is a medicine that contains the active substance chenodeoxycholic acid. This is a ‘primary bile acid’, which is a main component of bile (a fluid produced by the liver that helps to digest fats).

Chenodeoxycholic acid Leadiant is used to treat adults and children from one month of age with cerebrotendinous xanthomatosis. These patients cannot produce enough of the primary bile acid chenodeoxycholic acid due to genetic abnormalities that result in a lack of the liver enzyme sterol 27 hydroxylase. When primary bile acids are lacking, the body produces abnormal bile acids and other substances instead 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 Leadiant was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 16 December 2014.

Chenodeoxycholic acid Leadiant is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance. The reference medicine for Chenodeoxycholic acid Leadiant is Xenbilox. However, Xenbilox differs from Chenodeoxycholic acid Leadiant in that it is authorised for a different use (to dissolve cholesterol gallstones).

Chenodeoxycholic acid Leadiant can only be obtained with a prescription and treatment should be started and supervised by a doctor experienced in treating cerebrotendinous xanthomatosis or other diseases of primary bile acid production.

Chenodeoxycholic acid Leadiant is available as capsules (250 mg) which are taken 3 times a day at approximately the same time every day. The daily dose is chosen and adjusted during treatment for each patient depending on the patient’s age, liver function and levels of bile acids in their blood and urine. For small children and children who cannot swallow capsules, the contents of the capsules can be mixed with sodium bicarbonate solution 8.4% to make a liquid.

For further information, see the package leaflet.

Chenodeoxycholic acid is one of the main primary bile acids produced by the liver. The chenodeoxycholic acid contained in this medicine replaces the patient’s missing chenodeoxycholic acid. This helps to reduce the production of abnormal substances and contributes to the normal activity of bile in the digestive system, thereby relieving the symptoms of the disease.

Chenodeoxycholic acid Leadiant has been investigated in a study which looked at the records of 35 patients with cerebrotendinous xanthomatosis who received chenodeoxycholic acid for around 9 years. Among 23 patients for whom data on blood levels of bile acids were available, all had reductions in their levels (average reduction of 56–69 µmol/l). Among 14 patients for whom data on urine levels of bile acids were available, 79% (11 out of 14) had reduced levels at their most recent test. Most patients also had improvements in symptoms of the disease: all reported an improvement in diarrhoea, 89% of patients showed an improvement in their mental ability, 60% of patients showed improvement in mobility, and 85% and 77% of patients showed psychiatric improvement based on two different scores.

The side effects seen with Chenodeoxycholic acid Leadiant are constipation and abnormal liver test values; however their frequency could not be reliably estimated from the available limited data. They were mild or moderate in severity and did not last.

For the list of restrictions, see the package leaflet.

Chenodeoxycholic acid has been used to treat cerebrotendinous xanthomatosis for about 40 years, although it was not licensed for this use. However, due to the rarity of the disease, there are still limited data available on the use of the medicine. Nevertheless, studies have shown that the medicine benefits patients and has no significant side effects. The Agency’s Committee for Medicinal Products for Human Use (CHMP) therefore decided that Chenodeoxycholic acid Leadiant’s benefits are greater than its risks and recommended that it be approved for use in the EU.

Chenodeoxycholic acid Leadiant has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Chenodeoxycholic acid Leadiant due to the rarity of the disease. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.

Since Chenodeoxycholic acid Leadiant has been approved under exceptional circumstances, the company that markets this medicine will set up a registry to monitor the benefits and safety of the medicine.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Chenodeoxycholic acid Leadiant have been included in the summary of product characteristics and the package leaflet.

The European Commission granted a marketing authorisation valid throughout the European Union for Chenodeoxycholic acid sigma-tau on 10 April 2017. The name of the product was changed to Chenodeoxycholic acid Leadiant on 12 May 2017.

For more information about treatment with Chenodeoxycholic acid Leadiant, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Chenodeoxycholic acid Leadiant : EPAR - Summary for the public

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Product information

Chenodeoxycholic acid Leadiant : EPAR - Product Information

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Latest procedure affecting product information: S-22

20/07/2023

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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).

Chenodeoxycholic acid Leadiant : EPAR - All Authorised presentations

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Product details

Name of medicine
Chenodeoxycholic acid Leadiant (previously known as Chenodeoxycholic acid sigma-tau)
Active substance
Chenodeoxycholic acid
International non-proprietary name (INN) or common name
chenodeoxycholic acid
Therapeutic area (MeSH)
  • Xanthomatosis, Cerebrotendinous
  • Metabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code
A05AA01

Pharmacotherapeutic group

Bile and liver therapy

Therapeutic indication

Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults.

Authorisation details

EMA product number
EMEA/H/C/004061

Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Exceptional circumstances

This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Marketing authorisation holder
Leadiant GmbH

Liebherrstrasse 22
80538 München
Germany

Opinion adopted
21/07/2016
Marketing authorisation issued
10/04/2017
Revision
6

Assessment history

Chenodeoxycholic acid Leadiant : EPAR - Procedural steps taken and scientific information after authorisation

Chenodeoxycholic acid sigma-tau : EPAR - Public assessment report

CHMP summary of positive opinion for Chenodeoxycholic acid sigma-tau

Topics

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