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: 20 Mar 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 Inborn genetic disorders
  • Research Beta-thalassaemia

Most Recent Events

  • 14 Feb 2018 Agios Pharmaceuticals has issued as well as pending patents for Inborn error metabolic disorder therapeutics in USA and other countries worldwide
  • 14 Nov 2016 Early research in Beta-thalassaemia in USA
  • 26 Nov 2013 Preclinical trials in Inborn genetic disorders in USA (unspecified route)
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