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.
Treatment of ATTR amyloidosis
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Review of designation
Documents related to this orphan designation evaluation
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: