Pyrukynd - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation

mitapivat

Opinion

On 16 October 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Pyrukynd. The marketing authorisation holder for this medicinal product is Agios Netherlands B.V.

The CHMP adopted a new indication associated with a new strength, 100 mg film-coated tablets, as follows:

Pyrukynd is indicated in adults for the treatment of anaemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia (see section 5.1).

For information, the indication for Pyrukynd 5 mg, 20 mg and 50 mg film-coated tablets is as follows:

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

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

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Note: New text in bold.

CHMP post-authorisation summary of positive opinion for Pyrukynd (X-10-G)

Adopted Reference Number: EMA/329333/2025

Key facts

• Name of medicine:
  Pyrukynd
• EMA product number:
  EMEA/H/C/005540
• Active substance:
  mitapivat sulfate
• International non-proprietary name (INN) or common name:
  mitapivat
• Therapeutic area (MeSH):
  • Genetic Diseases, Inborn
  • Anemia, Hemolytic
• Anatomical therapeutical chemical (ATC) code:
  B06AX04
• Additional 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.

• Orphan:

  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.

• Marketing authorisation holder:
  Agios Netherlands B.V.
• Date of opinion:
  16/10/2025
• Status:
  Positive