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Mitapivat - Agios Pharmaceuticals

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Drug Profile

Mitapivat - Agios Pharmaceuticals

Alternative Names: AG 348; AG 348-sulfate-hydrate; Mitapivat-sulfate; PYRUKYND

Latest Information Update: 12 Dec 2024

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At a glance

  • Originator Agios Pharmaceuticals
  • Class Antianaemics; Benzene derivatives; Cyclopropanes; Piperazines; Quinolines; Small molecules; Sulfonamides
  • Mechanism of Action Pyruvate kinase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Sickle cell anaemia; Inborn error pyruvate metabolism disorders; Thalassaemia
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Inborn error pyruvate metabolism disorders
  • Preregistration Thalassaemia
  • Phase III Haemolytic anaemia
  • Phase II/III Sickle cell anaemia
  • Preclinical Hereditary spherocytosis

Most Recent Events

  • 08 Dec 2024 Preregistration for Thalassaemia in European Union (PO)
  • 08 Dec 2024 Preregistration for Thalassaemia in Saudi Arabia (PO)
  • 08 Dec 2024 Preregistration for Thalassaemia in United Arab Emirates (PO)

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