On 16 April 2018, orphan designation (EU/3/18/2011) was granted by the European Commission to Ultragenyx Germany GmbH, Germany, for burosumab for the treatment of phosphaturic mesenchymal tumour.
The medicinal product for treatment of phosphaturic mesenchymal tumour has been authorised in the EU as Crysvita since 25 July 2022.
Treatment of phosphaturic mesenchymal tumour
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Review of designation
The sponsorship was transferred to Kyowa Kirin Holdings B.V., The Netherlands, in February 2019.
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: