Kolbam - Cholic Acid

What is Kolbam - Cholic Acid and what is it used for?

Kolbam is a medicine containing cholic acid as an active ingredient.Cholic acid is a 'primary bile acid', a major component of bile (a liquid produced by the liver that aids in the digestion of fats).

Kolbam is authorized for the lifetime treatment of adults and children of at least one month of age who are unable to produce sufficient quantities of primary bile acids, such as cholic acid, due to specific genetic abnormalities resulting in a lack of following liver enzymes: sterol 27-hydroxylase, 2-methylacyl-CoA racemase or cholesterol 7Î ± -hydroxylase.

When primary bile acids are missing, the body produces abnormal bile acids that can damage the liver, with the risk of life-threatening liver failure. These disorders are known as "congenital defects of primary bile acid synthesis".

Because the number of patients with inborn defects of 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 - Cholic Acid?

Kolbam can only be obtained with a prescription and treatment should be started and supervised by a doctor who specializes in the diseases treated with Kolbam.

Kolbam is available in capsules (50mg and 250mg). The daily dose is decided and adjusted during the course of treatment based on the blood (blood) and urinary levels of bile acids and the liver function of each patient. The maximum daily dose should not exceed 15 mg per kilogram of body weight.

Kolbam should be taken at approximately the same time each day, in conjunction with a meal. In the case of young children who are unable to swallow the capsules, the contents can be mixed with infant formula, expressed breast milk, mashed potatoes or fruit puree.

For more information, see the package leaflet.

How does Kolbam - Cholic Acid work?

Cholic acid is one of the main primary bile acids produced by the liver. Cholic acid contained in Kolbam replaces the missing cholic acid in the patient's body. In this way, it helps reduce the production of abnormal bile acids and promotes the normal activity of bile in the digestive system, relieving the symptoms of the disorder.

What benefit has Kolbam - Cholic Acid shown during the studies?

Kolbam was studied in one main study involving 52 patients with inborn defects of primary bile acid synthesis, including 7 patients with lack of sterol 27-hydroxylase, 2-methylacyl-CoA racemase or cholesterol 7Î ± -hydroxylase. main efficacy parameters were changes in bile acid levels and liver function before and after treatment with Kolbam. Kolbam has been shown to reduce the amount of abnormal bile acids and improve liver function in the general population of patients with congenital defects. primary bile acid synthesis, and an improvement trend was observed in each individual enzyme deficiency, and these results were in line with clinical expectations and data from the medical literature.

What is the risk associated with Kolbam - Cholic Acid?

Kolbam side effects are usually mild to moderate in severity and transient in nature. The most common side effects (which may affect up to 1 in 10 people) are peripheral neuropathy (nerve damage in the hands and feet), diarrhea, nausea, acid reflux (reflux of stomach acid into the mouth), esophagitis (inflammation of the mouth). esophagus), jaundice (yellowing of the skin and eyes), skin problems (lesions) and malaise.

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

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

Why has Kolbam - Cholic Acid been 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 deficiency of the following liver enzymes: sterol 27-hydroxylase, 2-methylacyl-CoA racemase and cholesterol 7Î ± -hydroxylase Regarding safety, the undesirable effects appeared not serious and reversible.

Kolbam was authorized under "exceptional circumstances" because it was not possible to obtain complete information on Kolbam 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 Kolbam - Cholic Acid?

As Kolbam has been authorized under exceptional circumstances, the company that markets Kolbam will monitor the medicine's benefits and safety against a patient registry and provide annual updates.

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

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 added to the summary of product characteristics and 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 containing information on the correct and safe use of the medicine to all doctors who are expected to prescribe it.

More information about Kolbam - Cholic Acid

For more information about Kolbam therapy, read the package leaflet (included with the EPAR) or contact your doctor or pharmacist.

The information on Kolbam - Cholic Acid published on this page may be out of date or incomplete. For a correct use of this information, see the Disclaimer and useful information page.

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