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Active Substance: tocofersolan
Common Name: tocofersolan
ATC Code: A11HA08
Marketing Authorisation Holder: Orphan Europe S.A.R.L.
Active Substance: tocofersolan
Status: Authorised
Authorisation Date: 2009-07-24
Therapeutic Area: Vitamin E Deficiency Cholestasis
Pharmacotherapeutic Group: Vitamins

Therapeutic Indication

Vedrop is indicated in vitamin-E deficiency due to digestive malabsorption in paediatric patients suffering from congenital chronic cholestasis or hereditary chronic cholestasis, from birth (in term newborns) to 16 or 18 years of age, depending on the region.

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 deficiency (low vitamin-E levels). It is used in children up to the age of 18 years who have congenital or hereditary chronic cholestasis and who cannot absorb vitamin E from the gut. Congenital or hereditary chronic cholestasis is an inherited disease causing problems with the flow of bile from the liver to the gut. Bile is a fluid produced in the liver that helps to absorb fats from the gut.

The medicine can only be obtained with a prescription.

How is Vedrop used?

Treatment with Vedrop should be started and supervised by a doctor who has experience in treating patients with congenital or hereditary chronic cholestasis.

Vedrop is given by mouth, with or without water. The recommended daily dose is 0.34 ml per kilogram body weight. This dose should be adjusted depending on the amount of vitamin E in the child’s blood. This should be checked regularly.

How does Vedrop work?

Vitamin E is a natural substance that cannot be made by the body and is therefore needed in the diet. It has a number of actions in the body, including protecting the nervous system from damage. Because vitamin E is soluble in fats and not water, it can only be absorbed from the gut into the body alongside fat particles. Patients with cholestasis may have low vitamin-E levels because they have problems absorbing fats from the gut.

The active substance in Vedrop, tocofersolan, is vitamin E that has been made water soluble by attaching it to a chemical called polyethylene glycol. Tocofersolan can be absorbed from the gut in children who have difficulty absorbing fats and vitamin E from the diet. This can increase vitamin-E levels in the blood and help to prevent neurological deterioration (problems in 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 company presented information from the scientific literature, including the results of three studies in a total of 92 children and adolescents with chronic cholestasis who were given tocofersolan for about two years. The patients all had vitamin-E deficiency that was not responding to other vitamin-E treatments given by mouth. The main measures of effectiveness were the level of vitamin E in the blood and the number of children whose neurological symptoms improved or stayed the same.

The company also presented information on the use of Vedrop in patients with cystic fibrosis, but withdrew its application for this disease during the evaluation of the medicine.

What benefit has Vedrop shown during the studies?

The studies showed that Vedrop could correct vitamin-E levels in patients with chronic cholestasis and that it might improve or prevent neurological symptoms, especially in patients aged below three years.

What is the risk associated with Vedrop?

The most common side effect with Vedrop (seen in between 1 and 10 patients in 100) is diarrhoea. 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 ingredients. It must 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 for vitamin-E deficiency due to digestive malabsorption in paediatric patients suffering from congenital chronic cholestasis or hereditary chronic cholestasis, from birth (in term newborns) to 16 or 18 years of age, depending on the region. The Committee recommended that Vedrop be given marketing authorisation.

Vedrop has been authorised under ‘exceptional circumstances’. This means that because the disease is rare, it has not been possible to obtain complete information about Vedrop. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.

What information is still awaited for Vedrop?

The company that makes Vedrop will work with other companies to assess the possible effects of propylparaben (a preservative used in Vedrop) on the reproductive organs. The company will also set up a registry of patients with congenital or hereditary chronic cholestasis.

Other information about Vedrop

The European Commission granted a marketing authorisation valid throughout the European Union for Vedrop to Orphan Europe S.A.R.L. on 24 July 2009.

Source: European Medicines Agency

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