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Research programme: inborn metabolic disorder therapeutics - Agios Pharmaceuticals

Drug Profile

Research programme: inborn metabolic disorder therapeutics - Agios Pharmaceuticals

Alternative Names: IEM therapeutics - Agios Pharmaceuticals

Latest Information Update: 28 Jul 2018

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At a glance

  • Originator Agios Pharmaceuticals
  • Class Small molecules
  • Mechanism of Action Glycolysis modulators; Pyruvate kinase stimulants; Thyroid hormone binding protein stimulants
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Phenylketonuria
  • Research Beta-thalassaemia; Friedreich's ataxia
  • No development reported Inborn genetic disorders

Most Recent Events

  • 28 Jul 2018 No recent reports of development identified for preclinical development in Inborn genetic disorders in USA
  • 04 May 2018 Agios Pharmaceuticals plans an IND for Genetic disorders in the fourth quarter of 2018
  • 01 May 2018 Early research in Friedreich's ataxia in USA (unspecified route)
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