EU/3/19/2152: Orphan designation for the treatment of Stargardt's disease

3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid


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.

Key facts

Active substance
3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid
Intended use
Treatment of Stargardt's disease
Orphan designation status
EU designation number
Date of designation

TMC Pharma (EU) Limited
7A Durands Court
45 Parnell Street
Co. Waterford
X91 P381
Tel. + 353766705745

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

April 2023Please note that this product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.
May 2022The sponsor’s address was updated in May 2022.
March 2021The sponsor’s address was updated in March 2021.

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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