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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: 28 Apr 2026

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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

  • 24 Dec 2025 Alnylam Pharmaceuticals completes the phase III APOLLO-B trial for hereditary and wild-type ATTR Amyloidosis in Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, Czechia, Denmark, France, Hong Kong, Italy, Japan, Mexico, Netherlands, New Zealand, Poland, Portugal, South Korea, Sweden, Taiwan, the UK, US (IV, Infusion) (NCT03997383) (EudraCT2019-001458-24)
  • 19 Jun 2025 Launched for Amyloid polyneuropathy in Denmark (IV)
  • 13 Feb 2025 Brazilian Health Regulatory Agency approves Patisiran for the treatment of Amyloidosis with Cardiomyopathy

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