EU/3/10/823: Orphan designation for the treatment of familial chylomicronaemia



On 17 December 2010, orphan designation (EU/3/10/823) was granted by the European Commission to Dimensione Ricerca S.r.l., Italy, for lomitapide for the treatment of familial chylomicronaemia.

The sponsorship was transferred to Aegerion Pharmaceuticals, France, in February 2012 and subsequently to Aegerion Pharmaceuticals Limited, United Kingdom, in February 2015.

The sponsorship was transferred to Amryt Pharmaceuticals Designated Activity Company, Ireland, in March 2019.

Key facts

Active substance
Intended use
Treatment of familial chylomicronaemia
Orphan designation status
EU designation number
Date of designation

Amryt Pharmaceuticals Designated Activity Company
45 Mespil Road
Ballsbridge, Dublin 4
D04 W2F1
Co. Dublin

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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