EU/3/19/2192: Orphan designation for the treatment of Bardet-Biedl syndrome
Table of contents
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.
Treatment of Bardet-Biedl syndrome
|Orphan designation status||
|EU designation number||
|Date of designation||
Rhythm Pharmaceuticals Netherlands B.V.
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 .
|September 2022||The medicinal product was authorised in the EU as Imcivree|
|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.|
Documents related to this orphan designation evaluation
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: