Drug Profile


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

Latest Information Update: 17 Nov 2017

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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

  • Preregistration Amyloid polyneuropathy
  • Phase II Amyloidosis

Most Recent Events

  • 16 Nov 2017 Preregistration for Amyloid polyneuropathy in USA (IV)
  • 13 Nov 2017 Patisiran receives accelerated assessment status in Europe for Amyloidosis
  • 02 Nov 2017 Additional efficacy data from the phase III APOLLO trial in Amyloid polyneuropathy released by Alnylam
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