Drug Profile


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

Latest Information Update: 10 Sep 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

  • Phase III Amyloid polyneuropathy
  • Phase II Amyloidosis

Most Recent Events

  • 03 Jul 2017 Silence Therapeutics issues a claim in the UK High Courts of Justice (Patents Court) naming Alnylam UK Limited, Alnylam Pharmaceuticals and The Medicines Company UK Limited as defendants
  • 12 Jun 2017 Alnylam Pharmceuticals receives patent allowance for oligonucleotide-based transthyretin therapeutics in USA
  • 26 Apr 2017 Efficacy and adverse events data from the phase II OLE ALN-TTR02-003 trial in Amyloid polyneuropathy presented at the American Academy of Neurology (AAN) 2017 Annual Meeting
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