Drug Profile


Alternative Names: ALN AT3; ALN-AT3SC; SAR 439774

Latest Information Update: 20 Dec 2016

Price : $50

At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Antifibrinolytics; Antihaemorrhagics; Antisense RNA; Drug conjugates; Small interfering RNA
  • Mechanism of Action Antithrombin III inhibitors; Haemostasis stimulants; RNA interference
  • Orphan Drug Status

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

    Yes - Haemophilia A; Haemophilia B
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Haemophilia A; Haemophilia B

Most Recent Events

  • 03 Dec 2016 Safety and efficacy data from phase I trial and phase II open-label extension trial in Haemophila A and Haemophila B presented at the 58th Annual Meeting of the American Society of Haematology (ASH-2016)
  • 14 Nov 2016 Alnylam and Sanofi Genzyme agrees to co-develop and co-commercialise fitusiran for Haemophilia
  • 25 Jul 2016 Interim efficacy and adverse events data from a phase I trial in Haemophilia released by Alnylam Pharmaceuticals
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