Drug Profile


Alternative Names: ALN-AS1

Latest Information Update: 07 Jul 2017

Price : $50

At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action 5 aminolevulinate synthetase inhibitors
  • Orphan Drug Status

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

    Yes - Acute intermittent porphyria
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Acute intermittent porphyria

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
  • 26 Jun 2017 Alnylam Pharmaceuticals plans to initiate phase III programme for givosiran in Acute intermittent porphyria in late 2017
  • 26 Jun 2017 Interim adverse events and efficacy data from a phase I trial in Acute intermittent porphyria released by Alnylam
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