On 17 July 2012, orphan designation (EU/3/12/1023) was granted by the European Commission to Aptiv Solutions (UK) Limited, United Kingdom, for metreleptin for the treatment of Barraquer-Simons syndrome.
The sponsorship was transferred to Bristol-Myers Squibb / AstraZeneca EEIG, United Kingdom, in February 2014 then to AstraZeneca AB, Sweden, in April 2014 and subsequently to Aegerion Pharmaceuticals Limited, United Kingdom, in March 2015.
The sponsorship was transferred to Aegerion Pharmaceuticals B.V., The Netherland, in November 2017.
Metreleptin for treatment of Barraquer-Simons syndrome has been authorised in the EU as Myalepta since 30 July 2018.
The sponsorship was transferred to Amryt Pharmaceuticals Designated Activity Company, Ireland, in March 2020.
|Disease / condition||
Treatment of Barraquer-Simons syndrome
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.