EU/3/18/2081: Orphan designation for the treatment of ATTR amyloidosis

3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid


On 19 November 2018, orphan designation (EU/3/18/2081) was granted by the European Commission to Pharma Gateway AB, Sweden, for 3-(3-(3,5-dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid (also known as AG10) for the treatment of ATTR amyloidosis.

The sponsorship was transferred to Bridge Bio Europe B.V., Netherlands, in November 2021.

Key facts

Active substance
3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid
Intended use
Treatment of ATTR amyloidosis
Orphan designation status
EU designation number
Date of designation

Bridge Bio Europe B.V.
Herikerbergweg 238
1101 CM Amsterdam

Review of designation

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

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • 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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