On 17 July 2012, orphan designation (EU/3/12/1022) was granted by the European Commission to Aptiv Solutions (UK) Limited, United Kingdom, for metreleptin for the treatment of familial partial lipodystrophy.

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 familial partial lipodystrophy has been authorised in the EU as Myalepta since 30 July 2018.

Key facts

Active substance
Medicine name
Disease / condition
Treatment of familial partial lipodystrophy
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Myalepta at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF iconorphan medicine assessment report .

Sponsor's contact details

Aegerion Pharmaceuticals B.V.
Atrium Building, 8th Floor
Strawinskylaan 3127
1077 ZX Amsterdam
The Netherlands
Tel: +31 (0)88 560 9950

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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