On 4 August 2010, orphan designation (EU/3/10/764) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for everolimus for the treatment of tuberous sclerosis.

Everolimus has been authorised in the EU as Votubia since 2 September 2011.

The sponsorship was transferred to Novartis Europharm Limited, Ireland, in May 2018.

Key facts

Active substance
Medicine name
Disease / condition
Treatment of tuberous sclerosis
Date of first decision
EU designation number

Review of designation

During its meeting of 8-9 June 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/10/764 for Votubia (everolimus) as an orphan medicinal product for the treatment of tuberous sclerosis.

The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained.

Sponsor's contact details

Novartis Europharm Limited
Vista Building
Elm Park
Merrion Road
Dublin 4
Tel. +41 61 324 11 11 (Switzerland)

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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