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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: 24 Sep 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

  • 13 Sep 2024 Updated efficacy and adverse events data from the phase III ENERGIZE trial in Thalassaemia presented at the 29th Congress of the European Haematology Association (EHA-2024)
  • 01 Aug 2024 Efficacy and adverse events data from a phase III ACTIVATE-KidsT trial in Inborn error pyruvate metabolism disorders released by Agios Pharmaceuticals
  • 01 Aug 2024 Mitapivat licensed to NewBridge Pharmaceuticals in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates

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