Drug Profile


Alternative Names: ALN-TTR01

Latest Information Update: 04 Sep 2013

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Amyloid polyneuropathy
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Amyloidosis

Most Recent Events

  • 28 Aug 2013 Final efficacy data from a phase I trial in Amyloidosis released by Alnylam
  • 06 Aug 2012 Discontinued - Phase-I for Amyloidosis in France (IV)
  • 06 Aug 2012 Discontinued - Phase-I for Amyloidosis in Portugal (IV)
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