EU/3/16/1711: Orphan designation for the treatment of familial partial lipodystrophy

Volanesorsen sodium


On 14 July 2016, orphan designation (EU/3/16/1711) was granted by the European Commission to Ionis USA Ltd, United Kingdom, for volanesorsen sodium for the treatment of familial partial lipodystrophy.

The sponsorship was transferred to Akcea Therapeutics UK Ltd, United Kingdom, in March 2018 and subsequently to Akcea Therapeutics Ireland Limited, Ireland, in March 2019.

Key facts

Active substance
Volanesorsen sodium
Intended use
Treatment of familial partial lipodystrophy
Orphan designation status
EU designation number
Date of designation

Akcea Therapeutics Ireland Limited
St. James House
72 Adelaide Road
Dublin 2
Co. Dublin
D02 Y017

Review of designation

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

Update history

Date Update
January 2023 The sponsor's address was updated.

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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