Drug Profile


Alternative Names: ALN-AAT

Latest Information Update: 29 Mar 2018

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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

  • 15 Mar 2018 Alnylam Pharmaceuticals terminates a phase I/II trial due to observation of low incidence of asymptomatic, transiently elevated liver enzymes in a subset of study subjects in Alpha 1-antitrypsin deficiency in United Kingdom (SC) (NCT02503683)
  • 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
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