EU/3/17/1843: Orphan designation for the treatment of achondroplasia

Soluble recombinant human fibroblast growth factor receptor 3


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
EU designation number
Date of designation

Pfizer Europe MA EEIG
Boulevard de la Plaine 17
1050 Bruxelles
Brussels-Capital Region

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it 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:

  • 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:

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