Drug Profile


Alternative Names: ALN-AS1

Latest Information Update: 02 Jun 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

  • 31 May 2017 Givosiran receives Breakthrough Therapy status for Acute intermittent porphyria in USA
  • 01 Mar 2017 Givosiran receives Priority Medicine (PRIME) status for Acute intermittent porphyria in European Union
  • 03 Dec 2016 Efficacy and adverse events data from a phase I trial in Acute intermittent porphyria presented at the 58th Annual Meeting of the American Society of Hematology (ASH-2016)
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