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Patisiran - Alnylam Pharmaceuticals

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Drug Profile

Patisiran - Alnylam Pharmaceuticals

Alternative Names: ALN-18328; ALN-TTR02; GENZ-438027; ONPATTRO; Onpattro; Patisiran sodium - Alnylam Pharmaceuticals; patisiran-LNP; SAR-438037

Latest Information Update: 21 Feb 2025

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action Prealbumin expression inhibitors; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Amyloid polyneuropathy; Amyloidosis
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Amyloid polyneuropathy; Amyloidosis

Most Recent Events

  • 13 Feb 2025 Brazilian Health Regulatory Agency approves Patisiran for the treatment of Amyloidosis with Cardiomyopathy
  • 30 Apr 2024 Alnylam Pharmaceuticals expands its agreement with Medison Pharma to commercialise Patisiran, in additional Latin America and APAC markets including Australia
  • 09 Oct 2023 Alnylam Therapeutics receives complete response letter from the US FDA for patisiran for Cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis

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