Givosiran

Drug Profile

Givosiran

Alternative Names: ALN-AS1

Latest Information Update: 15 Feb 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
  • 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Acute intermittent porphyria

Most Recent Events

  • 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)
  • 01 Oct 2016 Phase-I/II clinical trials in Acute intermittent porphyria in USA, Sweden, United Kingdom (SC) (NCT02949830)
  • 07 Sep 2016 ALN AS1 receives Orphan Drug status for Acute intermittent porphyria in USA
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