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Givosiran - Alnylam Pharmaceuticals

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Drug Profile

Givosiran - Alnylam Pharmaceuticals

Alternative Names: ALN-AS1; GIVLAARI

Latest Information Update: 06 May 2024

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Amides; Amino sugars; Drug conjugates; 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 - Hepatic-porphyria
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Hepatic-porphyria

Most Recent Events

  • 30 Apr 2024 Alnylam Pharmaceuticals expands its agreement with Medison Pharma to commercialise Givosiran in additional Latin America and APAC markets including Australia
  • 28 Nov 2023 Registered for Hepatic-porphyria (In adolescents, In adults) in Australia (SC)
  • 21 Jun 2023 Final efficacy and adverse event data from a phase I/II trial in Hepatic porphyria presented at the European Association for the Study of the Liver Congress 2023 (EASL-2023)

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