This medicine is authorised for use in the European Union.


Pyrukynd is a medicine used to treat adults with pyruvate kinase deficiency (PKD), an inherited disease that causes red blood cells to break down faster than normal.

PKD is rare, and Pyrukynd was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 22 April 2020. Further information on the orphan designation can be found here:

Pyrukynd contains the active substance mitapivat.

This EPAR was last updated on 05/12/2022

Authorisation details

Product details
Agency product number
Active substance
mitapivat sulfate
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
  • Genetic Diseases, Inborn
  • Anemia, Hemolytic
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Agios Netherlands B.V.
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Zuidplein 36
1077XV Amsterdam

Product information

Pyrukynd - EMEA/H/C/005540 -

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

Pharmacotherapeutic group

Other hematological agents

Therapeutic indication

Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients (see section 4.4).

Assessment history

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