Research programme: rare genetic disease therapeutics - Alexion Pharmaceuticals/Blueprint Medicines
Latest Information Update: 29 Aug 2015
At a glance
- Originator Blueprint Medicines
- Mechanism of Action Protein kinase modulators
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
- Research Inborn genetic disorders