Drug Profile


Alternative Names: ALN-TTR02; GENZ-438027; LNP-siTTR; SAR-438037

Latest Information Update: 25 Mar 2017

Price : $50

At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Oligonucleotides; 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

  • Phase III Amyloid polyneuropathy
  • Phase II Amyloidosis

Most Recent Events

  • 18 Oct 2016 Alnylam Pharmaceuticals initiates an expanded-access programme for hereditary transthyretin-mediated Amyloidosis with polyneuropathy in USA (IV, Infusion) (NCT02939820)
  • 01 Aug 2016 Alnylam completes a phase II extension trial for Amyloidosis in USA, Brazil, France, Germany, Portugal, Spain and Sweden (NCT01961921)
  • 05 Jul 2016 Efficacy and adverse events data from the phase II OLE trial in Amyloid polyneuropathy released by Alnylam Pharmaceuticals
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