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Mitapivat - Agios Pharmaceuticals

Drug Profile

Mitapivat - Agios Pharmaceuticals

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

Latest Information Update: 12 Mar 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
  • Phase III Haemolytic anaemia; Thalassaemia
  • Phase II/III Sickle cell anaemia
  • Preclinical Hereditary spherocytosis

Most Recent Events

  • 29 Feb 2024 Agios Pharmaceuticals plans a phase II trial for Sickle cell anaemia and Kidney disorders (In adolescents, In adults, In the elderly) (PO) in June 2024 (NCT06286046)
  • 28 Feb 2024 No recent reports of development identified for phase-I development in Inborn-error-pyruvate-metabolism-disorders(In volunteers) in USA (PO, Granules)
  • 15 Feb 2024 Agios Pharmaceuticals announces intention to launch Mitapivat in the US for the treatment of thalassemia in 2025
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