EU/3/18/2011: Orphan designation for the treatment of phosphaturic mesenchymal tumour



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.

Key facts

Active substance
Intended use
Treatment of phosphaturic mesenchymal tumour
Orphan designation status
EU designation number
Date of designation

Kyowa Kirin Holdings B.V.
Bloemlaan 2
2132NP Hoofddorp
The Netherlands
The Netherlands

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Crysvita at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the PDF icon orphan medicine assessment report .

Update history

The sponsorship was transferred to Kyowa Kirin Holdings B.V., The Netherlands, in February 2019.

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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