Drug Profile


Alternative Names: ALN-60212; ALN-PCSsc; PCSK9si

Latest Information Update: 14 Sep 2017

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Developer Alnylam Pharmaceuticals; The Medicines Company
  • Class Amino sugars; Antihyperlipidaemics; Drug conjugates; Small interfering RNA
  • Mechanism of Action PCSK9 protein inhibitors; RNA interference
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Hypercholesterolaemia

Most Recent Events

  • 28 Aug 2017 Alnylam Pharmaceuticals announces intention to submit MAA and NDA applications for Hypercholesterolaemia in the second half of 2019
  • 28 Aug 2017 Interim efficacy and adverse events data from the phase II ORION-1 trial in Hypercholesterolaemia released by Alnylam Pharmaceuticals
  • 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
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