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
Disease / condition
Treatment of Stargardt's disease
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

TMC Pharma (EU) Limited
The Black Church
St. Mary's Place
Dublin D07 P4AX
Tel. +353 76 670 5745

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.

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