EU/3/18/2101: Orphan designation for the treatment of leptin receptor deficiency



Setmelanotide has been authorised in the EU as Imcivree since 16 July 2021.

On 19 November 2018, orphan designation (EU/3/18/2101) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for setmelanotide for the treatment of leptin receptor deficiency.

Key facts

Active substance
Intended use
Treatment of leptin receptor deficiency
Orphan designation status
EU designation number
Date of designation

Rhythm Pharmaceuticals Netherlands B.V.
Radarweg 29
1043 NX Amsterdam
tel: +31 20 854 6071

Review of designation

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

More information is available in the orphan medicine assessment report.

Update history

Date Update
July 2022 The sponsor's address was updated.
October 2021 The sponsorship was transferred to Rhythm Pharmaceuticals Netherlands B.V. in October 2021.
November 2020 The sponsorship was transferred to Rhythm Pharmaceuticals Limited, Ireland, in November 2020.
March 2019 The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in March 2019.

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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