Vedrop - tocofersolan

What is Vedrop?

Vedrop is an oral solution containing the active substance tocofersolan.

What is Vedrop used for?

Vedrop is indicated to treat or prevent vitamin E deficiencies (low levels of vitamin E). The medicine is used in children and adolescents up to 18 years of age with chronic congenital or hereditary cholestasis, in which the intestine does not absorb adequate amounts of vitamin E. Chronic congenital or hereditary cholestasis is an inherited disease for which the bile cannot flow from the liver to the intestine. Bile is a liquid produced in the liver that serves to absorb fats from the intestine.

The medicine can only be obtained with a prescription.

How is Vedrop used?

Treatment with Vedrop should be started and supervised by a doctor experienced in managing patients with chronic congenital or hereditary cholestasis.

Vedrop is taken orally, with or without water. The recommended daily dose is 0.34 ml per kilogram of body weight. The dose should be adjusted according to the level of vitamin E in the child's blood. This value should be checked regularly.

How does Vedrop work?

Vitamin E is a natural substance that, not being produced by the body, must be supplemented by food. It performs numerous functions within the body, including that of protecting the nervous system. Since vitamin E dissolves in fats and not in water, it is absorbed by the body only through the intestine, along with fat particles. In patients with cholestasis, low levels of vitamin E can be due to malabsorption of fat from the intestine.

The active ingredient in Vedrop, tocofersolan, consists of a form of vitamin E that is made soluble in water by hooking it to a chemical called polyethylene glycol. Tocofersolan can be absorbed from the intestine in children who have difficulty absorbing fats and vitamin E from the diet. This can increase the levels of vitamin E in the blood and help prevent neurological deterioration (disorders within the nervous system) due to vitamin E deficiency.

How has Vedrop been studied?

The effects of Vedrop were first tested in experimental models before being studied in humans.

To support the use of Vedrop, the pharmaceutical company presented information from the scientific literature, including the results of three studies conducted on a total of 92 children and adolescents with chronic cholestasis, who had been given tocofersolan for a period of about two years . The patients all had vitamin E deficiency, and had not responded to other oral vitamin E-based treatments. The main efficacy criteria were based on the level of vitamin E present in the blood and on the number of subjects whose neurological symptoms had improved or remained stable.

Initially, the company presented information concerning the use of Vedrop even for patients with cystic fibrosis, however during the evaluation of the medicine the application was withdrawn with reference to this disorder.

What benefit has Vedrop shown during the studies?

Studies have shown that Vedrop corrects vitamin E levels in patients with chronic cholestasis and that it can improve or prevent neurological symptoms, particularly in patients under the age of three.

What is the risk associated with Vedrop?

The most common side effect reported with Vedrop (seen in between 1 and 10 patients out of 100) is diarrhea. For the full list of all side effects reported with Vedrop, see the Package Leaflet.

Vedrop should not be used in people who may be hypersensitive (allergic) to tocofersolan or any of the other components. The medicine should not be used in premature babies.

Why has Vedrop been approved?

The Committee for Medicinal Products for Human Use (CHMP) has determined that Vedrop's benefits outweigh the risks in vitamin E deficiencies due to digestive malabsorption in pediatric patients suffering from chronic congenital cholestasis or hereditary chronic cholestasis, from birth (newborns to term) up to 16 or 18 years of age, depending on the region. The Committee therefore recommended that Vedrop be given marketing authorization.

Vedrop was authorized in "exceptional circumstances". This means that, as the disease is rare, it has not been possible to obtain complete information on Vedrop. The European Medicines Agency reviews each year the new information that may have become available and, if necessary, this summary will be updated.

What information is still awaited for Vedrop?

The company that produces Vedrop will work with other pharmaceutical companies to evaluate the possible effects of propylparaben (a preservative contained in Vedrop) on reproductive organs. The company also intends to establish a register of patients suffering from congenital or hereditary chronic cholestasis.