Research programme: inborn metabolic disorder therapeutics - Agios PharmaceuticalsAlternative Names: IEM therapeutics - Agios Pharmaceuticals
Latest Information Update: 15 Nov 2016
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.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity Yes
Highest Development Phases
- Preclinical Inborn genetic disorders
- Research Beta-thalassaemia