Vedrop - tocofersolan

What is Vedrop?

Vedrop is an oral solution that contains the active substance tocofersolan.

What is Vedrop used for?

Vedrop is used to treat or prevent vitamin E deficiencies (low vitamin E levels). The medicine is used in children and adolescents up to 18 years of age with congenital or hereditary chronic 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 is used to absorb fat from the intestine.
The medicine can only be obtained with a prescription.

How is Vedrop used?

Treatment with Vedrop should be initiated and supervised by a physician experienced in the management of patients with congenital or hereditary chronic 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 which, not being produced by the body, must be supplemented by food. It performs many 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, together with fat particles. In patients with cholestasis, low levels of vitamin E may be due to a malabsorption of fat by the intestine.
The active ingredient in Vedrop, tocofersolan, consists of a form of vitamin E that is made soluble in water by attaching it to a chemical called polyethylene glycol. Tocofersolan can be absorbed from the intestine in children who have difficulty absorbing fat and vitamin E from the diet. This can increase vitamin E levels 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.
In support of the use of Vedrop, the pharmaceutical company presented information from the scientific literature, including the results of three studies involving a total of 92 children and adolescents with chronic cholestasis who had been given tocofersolan over a period of approximately two years. The patients all had vitamin E deficiency, and did not respond to other oral vitamin E treatments. The main efficacy criteria were based on the level of vitamin E in the blood and the number of subjects whose neurological symptoms improved. or remained stable.
Initially, the company submitted information concerning the use of Vedrop also for patients with cystic fibrosis, however during the evaluation of the medicine the application was withdrawn in relation 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 1 to 10 patients out of a total of 100) is diarrhea. For the full list of 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 ingredients. The medicine should not be used in premature babies.

Why has Vedrop been approved?

The Committee for Medicinal Products for Human Use (CHMP) decided that Vedrop's benefits are greater than its risks in vitamin E deficiency due to digestive malabsorption in pediatric patients suffering from Congenital Chronic Cholestasis or Chronic Hereditary Cholestasis, from birth (neonates to term) up to 16 or 18 years of age, depending on the region. The Committee therefore recommended the granting of the marketing authorization for Vedrop.
Vedrop was authorized under "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 every year any 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 makes Vedrop will work with other pharmaceutical companies to evaluate the possible effects of propylparaben (a preservative contained in Vedrop) on the reproductive organs. The company also intends to establish a registry of patients with congenital or hereditary chronic cholestasis.

Other information about Vedrop:

On 24 July 2009, the European Commission released Orphan Europe S.A.R.L.
a "marketing authorization" for Vedrop, valid throughout the European Union.
For the full version of Vedrop EPAR click here.

Last update of this summary: 06-2009

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