Kolbam - Colic Acid

What is Kolbam 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 main component of bile (a liquid produced by the liver that favors the digestion of fats).

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

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

Since the number of patients with congenital defects of primary bile acid synthesis is low, this condition is considered "rare" and Kolbam was designated as an "orphan medicine" (a medicine used in rare diseases) on 28 October 2009.

How is Kolbam used - Colic Acid?

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

Kolbam is available as capsules (50 mg and 250 mg). The daily dose is decided and adjusted during treatment based on blood levels (in the 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 every day, in conjunction with a meal. In the case of small children who are unable to swallow the capsules, the contents can be mixed with infant formula, extracted breast milk, mashed potatoes or fruit puree.

For more information, see the package leaflet.

How does Kolbam - Colic Acid work?

Cholic acid is one of the main primary bile acids produced by the liver. The cholic acid in Kolbam replaces the missing cholic acid in 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 disorder.

What benefit has Kolbam - Colic acid shown during the studies?

Kolbam has been studied in one main study involving 52 patients with congenital defects of primary bile acid synthesis, including 7 patients with a lack of sterol 27-hydroxylase, 2-methylacyl-CoA racemase or 7Î ± -hydroxylase cholesterol. The 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 to improve liver function in the general population of patients with congenital defects of primary bile acid synthesis; furthermore, a tendency to improvement was observed in each individual enzyme deficiency. These results were in line with clinical expectations and with data from the medical literature.

What is the risk associated with Kolbam - Colic Acid?

The side effects of Kolbam are generally mild to moderate and of a transient nature. The most common side effects (which may affect up to 1 in 10 people) are peripheral neuropathy (damage to the hands and feet nerves), diarrhea, nausea, acid reflux (reflux of gastric acids in the oral cavity), esophagitis (inflammation of the esophagus), jaundice (yellowing of the skin and eyes), skin problems (lesions) and malaise.

Kolbam should not be used in combination with phenobarbital (a medicine for the treatment of epilepsy).

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

Why has Kolbam - Colic 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. As for safety, the undesirable effects seemed not serious and reversible.

Kolbam was authorized in "exceptional circumstances" because complete information on Kolbam 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 Kolbam - Acico Colico?

Because Kolbam has been authorized in exceptional circumstances, the company that markets Kolbam will monitor the benefits and safety of the medicine based on a patient registry and provide annual updates.

What measures are being taken to ensure the safe and effective use of Kolbam - Colic 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 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 containing information on the correct and safe use of the medicine to all doctors who are intended to prescribe it.