EU/3/19/2152: Orphan designation for the treatment of Stargardt's disease
Table of contents
On 24 April 2019, orphan designation (EU/3/19/2152) was granted by the European Commission to TMC Pharma (EU) Limited, Ireland, for 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-pyrazol-1-YL)propanoic acid (also known as STG‑001) for the treatment of Stargardt's disease.
Treatment of Stargardt's disease
|Orphan designation status||
|EU designation number||
|Date of designation||
TMC Pharma (EU) Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
|April 2023||Please note that this product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.|
|May 2022||The sponsor’s address was updated in May 2022.|
|March 2021||The sponsor’s address was updated in March 2021.|
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: