On 21 March 2018, orphan designation (EU/3/18/1997) was granted by the European Commission to QRC Consultants Ltd, United Kingdom, for miransertib for the treatment of Proteus syndrome.

The sponsorship was transferred to QRC Ireland, Ireland, in September 2018.

The sponsorship was transferred to Merck Sharp & Dohme B.V., Netherlands in December 2020.

Key facts

Active substance
Disease / condition
Treatment of Proteus syndrome
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Merck Sharp & Dohme B.V.
Waarderweg 39
2031 BN Haarlem
The Netherlands
Tel. +31 2351 53260

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.

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