EU/3/20/2270: Orphan designation for the treatment of pyruvate kinase deficiency
Table of contents
On 22 April 2020, orphan designation EU/3/20/2270 was granted by the European Commission to Agios Netherlands B.V., the Netherlands, for mitapivat sulfate (also known as AG-348) for the treatment of pyruvate kinase deficiency.
The medicinal product has been authorised in the EU as Pyrukynd since 9 November 2022.
Treatment of pyruvate kinase deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
Agios Netherlands B.V.
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Pyrukynd at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the orphan medicine assessment report
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: