Givosiran - Alnylam Pharmaceuticals

Drug Profile

Givosiran - Alnylam Pharmaceuticals

Alternative Names: ALN-AS1

Latest Information Update: 26 Apr 2018

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Oligonucleotides; Pyrrolidines; 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

  • 02 Aug 2018 Alnylam Pharmaceuticals anticipates to launch givosiran in Acute intermittent porphyria in 2019, assuming regulatory approvals
  • 03 May 2018 Alnylam Pharmaceuticals announces intention to submit MAA to the EMA for Acute intermittent porphyria in 2019
  • 23 Apr 2018 Alnylam Pharmaceuticals plans a phase I trial for Acute Intermittent Porphyria in Sweden , (NCT03505853)
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