Givosiran - Alnylam Pharmaceuticals

Drug Profile

Givosiran - Alnylam Pharmaceuticals

Alternative Names: ALN-AS1

Latest Information Update: 10 Nov 2017

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action 5 aminolevulinate synthetase inhibitors; RNA interference
  • 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 III Acute intermittent porphyria

Most Recent Events

  • 07 Nov 2017 Phase-III clinical trials in Acute intermittent porphyria (In adolescents, In the elderly, In adults) in USA (SC) (NCT03338816)
  • 07 Sep 2017 Alnylam announces intention to submit NDA to the US FDA for Acute intermittent porphyria at the end of 2018
  • 07 Sep 2017 Adverse events and efficacy data from a phase I trial in Acute intermittent porphyria released by Alnylam
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