EU/3/17/1843: Orphan designation for the treatment of achondroplasia
Soluble recombinant human fibroblast growth factor receptor 3
Table of contents
Overview
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.
Key facts
Active substance |
Soluble recombinant human fibroblast growth factor receptor 3
|
Intended use |
Treatment of achondroplasia
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/17/1843
|
Date of designation |
27/02/2017
|
Sponsor |
Pfizer Europe MA EEIG |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.
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: