On 27 February 2017, orphan designation (EU/3/17/1843) was granted by the European Commission to TherAchon SAS, France, for soluble recombinant human fibroblast growth factor receptor 3 (also known as rhFGFR3) for the treatment of achondroplasia.
The sponsorship was transferred to Pfizer Europe MA EEIG in November 2019.
Soluble recombinant human fibroblast growth factor receptor 3
Treatment of achondroplasia
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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:
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: