EU/3/19/2192: Orphan designation for the treatment of Bardet-Biedl syndrome



On 21 August 2019, orphan designation EU/3/19/2192 was granted by the European Commission to TMC Pharma (EU) Limited, Ireland, for setmelanotide for the treatment of Bardet Biedl syndrome.

The medicinal product for treatment of Bardet Biedl syndrome has been authorised in the EU as Imcivree since 2 September 2022.

Key facts

Active substance
Intended use
Treatment of Bardet-Biedl syndrome
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 PDF icon orphan medicine assessment report .

Update history

September 2022The medicinal product was authorised in the EU as Imcivree
July 2022The sponsor's address was updated.
October 2021The sponsorship was transferred to Rhythm Pharmaceuticals Netherlands B.V. in October 2021.
November 2020The sponsorship was transferred to Rhythm Pharmaceuticals Limited, Ireland, in November 2020.

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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