Drug Profile


Alternative Names: ALN-AAT

Latest Information Update: 10 Sep 2017

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action Alpha 1 antitrypsin inhibitors; RNA interference
  • 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

  • Phase I/II Alpha 1-antitrypsin deficiency

Most Recent Events

  • 12 Jun 2017 Alnylam Pharmceuticals receives patent allowance for GalNac conjugation technique in USA
  • 28 Sep 2016 Pharmacodynamics and safety data from a phase I/II trial in Alpha 1-antitrypsin deficiency released by Alnylam Pharmaceuticals
  • 04 Aug 2015 ALN AAT is covered by patents in USA, Europe, Japan, Australia, New Zealand, South Korea and China
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