EU/3/17/1910: Orphan designation for the treatment of tuberous sclerosis



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.

Key facts

Active substance
Medicine name
Intended use
Treatment of tuberous sclerosis
Orphan designation status
EU designation number
Date of designation

Plusultra Pharma GmbH
Fritz-Vomfelde-Strasse 36
40547 Duesseldorf 
North Rhine-Westphalia

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

Patients' organisations

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:

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