Kolbam

cholic acid

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This is a summary of the European public assessment report (EPAR) for Kolbam. 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 Kolbam.

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

What is Kolbam and what is it used for?

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

Kolbam is authorised for the life-long treatment of adults and children from one month of age who cannot produce enough primary bile acids such as cholic acid due to certain genetic abnormalities that result in a lack of the following liver enzymes: sterol 27-hydroxylase; 2-methylacyl-CoA racemase; or cholesterol 7a-hydroxylase.

When primary bile acids are lacking, the body produces abnormal bile acids instead, which can damage the liver potentially leading to life-threatening liver failure. The condition is known as ‘inborn errors in primary bile acid synthesis’.

Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Kolbam was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 October 2009.

How is Kolbam used?

Kolbam can only be obtained with a prescription and treatment should be started and supervised by a doctor specialised in the diseases Kolbam is used to treat.

Kolbam is available as capsules (50 and 250 mg). The daily dose is chosen and adjusted during treatment for each patient depending on the patient’s levels of bile acids in their blood and urine and their liver function. The maximum daily dose should not exceed 15 mg per kilogram body weight.

Kolbam should be taken every day at approximately the same time, with a meal. For small children who cannot swallow capsules, the contents can be mixed in with infant formula, expressed breast milk, mashed potatoes or fruit puree.

For more information, see the package leaflet.

How does Kolbam work?

Cholic acid is one of the main primary bile acids produced by the liver. The cholic acid contained in Kolbam replaces the patient’s missing cholic acid. This helps to reduce 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.

What benefits of Kolbam have been shown in studies?

Kolbam was investigated in one main study involving 52 patients with inborn errors in primary bile acid synthesis, including 7 patients who lack either sterol 27-hydroxylase, 2-methylacyl-CoA racemase or cholesterol 7a-hydroxylase. The main measures of effectiveness were changes in bile acid levels and liver function before and after treatment with Kolbam. The efficacy of Kolbam for the authorised indications was established based on the results of this study. This was consistent with clinical expectations and literature data.

What are the risks associated with Kolbam?

Side effects with Kolbam are generally mild to moderate in severity and transitory. The most common side effects (which may affect up to 1 in 10 people) are peripheral neuropathy (nerve damage in the hands and feet), diarrhoea, nausea (feeling sick), acid reflux (stomach acid flowing up into the mouth), oesophagitis (inflammation of the food pipe), jaundice (yellowing of the skin and eyes), skin problems (lesions) and malaise (feeling unwell).

Kolbam must not be used in combination with phenobarbital (a medicine for epilepsy).

For the full list of all side effects and restrictions, see the package leaflet.

Why is Kolbam approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Kolbam’s benefits are greater than its risks and recommended that it be approved for use in the EU. The CHMP concluded that Kolbam has beneficial effects in patients with deficiencies in the following liver enzymes: sterol 27-hydroxylase; 2-methylacyl-CoA racemase; and cholesterol 7a-hydroxylase. Regarding its safety, the side effects appeared to be non-serious and reversible.

Kolbam has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Kolbam 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.

What information is still awaited for Kolbam?

Since Kolbam has been approved under exceptional circumstances, the company that markets Kolbam will monitor the benefits and safety of Kolbam from a patient registry and provide yearly updates.

What measures are being taken to ensure the safe and effective use of Kolbam?

A risk management plan has been developed to ensure that Kolbam 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 Kolbam, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Kolbam will provide educational material with information on the correct and safe use of Kolbam to all doctors expected to prescribe this medicine.

Further information can be found in the summary of the risk management plan.

Other information about Kolbam

The European Commission granted a marketing authorisation valid throughout the European Union for Kolbam on 20 November 2015.

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

Name Language First published Last updated
Kolbam : EPAR - Summary for the public BG = bălgarski 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public ES = español 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public CS = čeština 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public DA = dansk 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public DE = Deutsch 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public ET = eesti keel 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public EL = elliniká 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public EN = English 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public FR = français 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public IT = italiano 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public LV = latviešu valoda 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public LT = lietuvių kalba 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public HU = magyar 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public MT = Malti 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public NL = Nederlands 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public PL = polski 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public PT = português 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public RO = română 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public SK = slovenčina 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public SL = slovenščina 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public FI = suomi 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public SV = svenska 09/12/2015 15/02/2016
Kolbam : EPAR - Summary for the public HR = Hrvatski 09/12/2015 15/02/2016
Name Language First published Last updated
Kolbam : EPAR - Risk-management-plan summary (English only) 09/12/2015 04/02/2016

This EPAR was last updated on 15/02/2016 .

Authorisation details

Product details

Product details for Kolbam
NameKolbam
Agency product numberEMEA/H/C/002081
Active substance

cholic acid

International non-proprietary name (INN) or common name

cholic acid

Therapeutic area Metabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code A05AA03
Additional monitoring

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

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Exceptional Circumstances

There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Kolbam
Marketing-authorisation holder

Retrophin Europe Ltd

Revision10
Date of issue of marketing authorisation valid throughout the European Union20/11/2015

Contact address:

Retrophin Europe Ltd
Palmerston House
Fenian Street
Dublin 2
Ireland

Product information

Product information

11/01/2016  Kolbam -EMEA/H/C/002081 -IAIN/0014

Name Language First published Last updated
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016
Kolbam : EPAR - Product Information EN = English 03/12/2015 10/02/2016

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  
Kolbam : EPAR - All Authorised presentations EN = English 09/12/2015  

Pharmacotherapeutic group

Bile and liver therapy

Therapeutic indication

Cholic Acid FGK is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects:

  • sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency;
  • 2- (or alpha-) methylacyl-CoA racemase (AMACR) deficiency;
  • cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Kolbam : EPAR - Procedural steps taken and scientific information after authorisation EN = English 11/09/2014 10/02/2016

Initial marketing-authorisation documents

Name Language First published Last updated
Kolbam : EPAR - Public assessment report EN = English 09/12/2015  
CHMP summary of opinion for Kolbam EN = English 25/09/2015 14/10/2015
CHMP summary of positive opinion Kolbam EN = English 25/09/2015