EU/3/12/1023: Orphan designation for the treatment of Barraquer-Simons syndrome

Metreleptin

Overview

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.

Key facts

Active substance
Metreleptin
Medicine name
Myalepta
Intended use
Treatment of Barraquer-Simons syndrome
Orphan designation status
Positive
EU designation number
EU/3/12/1023
Date of designation
17/07/2012
Sponsor

Amryt Pharmaceuticals Designated Activity Company
45 Mespil Road
Ballsbridge, Dublin 4
D04 W2F1
Co. Dublin
E-mail: medinfo@amrytpharma.com

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 icon orphan medicine assessment report .

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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