EU/3/17/1910: Orphan designation for the treatment of tuberous sclerosis
Table of contents
On 23 August 2017, orphan designation (EU/3/17/1910) was granted by the European Commission to Best Regulatory Consulting Ltd, United Kingdom, for sirolimus for the treatment of tuberous sclerosis.
The sponsorship was transferred to FGK Representative Service GmbH, Germany, in March 2019.
The sponsorship was transferred to Plusultra Pharma GmbH, Germany, in November 2021.
Treatment of tuberous sclerosis
|Orphan designation status||
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Plusultra Pharma GmbH
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Documents related to this orphan designation evaluation
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: