Drug Profile


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

Latest Information Update: 20 May 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

  • 26 Apr 2017 Efficacy and adverse events data from the phase II APOLLO-OLE trial in Amyloid polyneuropathy presented at the American Academy of Neurology (AAN) 2017 Annual Meeting
  • 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)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top