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.
Treatment of tuberous sclerosis
|Orphan designation status||
|EU designation number||
|Date of designation||
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.
|September 2023||Please note that this product was withdrawn from the Community Register of designated orphan medicinal products at the end of the 12-year period of market exclusivity.|
|May 2018||The sponsorship was transferred to Novartis Europharm Limited, Ireland.|
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of addition of new indications to the marketing authorisation: Votubia (everolimus) for the treatment of tuberous sclerosis (PDF/111.28 KB) (updated)
First published: 10/10/2011
Last updated: 15/09/2023
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: