EU/3/20/2270: Orphan designation for the treatment of pyruvate kinase deficiency

mitapivat sulfate


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.

Key facts

Active substance
mitapivat sulfate
Intended use
Treatment of pyruvate kinase deficiency
Orphan designation status
EU designation number
Date of designation

Agios Netherlands B.V. 
Regus Wtc 
Zuidplein 36 
1077 XV Amsterdam  

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 PDF icon orphan medicine assessment report

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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