Research programme: rare genetic disease therapeutics - Alexion Pharmaceuticals/Blueprint Medicines

Drug Profile

Research programme: rare genetic disease therapeutics - Alexion Pharmaceuticals/Blueprint Medicines

Latest Information Update: 29 Aug 2015

Price : $50

At a glance

  • Originator Blueprint Medicines
  • Class
  • 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.

    No
  • 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Research Inborn genetic disorders

Most Recent Events

  • 03 Mar 2015 Early research in Inborn genetic disorders in USA (unspecified route)
  • 03 Mar 2015 Protein kinase modulators licensed to Alexion pharmaceuticals worldwide for the treatment of Inborn genetic disorders
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