Patisiran - Alnylam Pharmaceuticals

Drug Profile

Patisiran - Alnylam Pharmaceuticals

Alternative Names: ALN-TTR02; GENZ-438027; SAR-438037

Latest Information Update: 15 Feb 2018

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At a glance

  • Originator Alnylam Pharmaceuticals
  • Class Oligonucleotides; Small interfering RNA
  • Mechanism of Action 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
  • New Molecular Entity Yes

Highest Development Phases

  • Preregistration Amyloidosis
  • Phase III Amyloid polyneuropathy

Most Recent Events

  • 08 Feb 2018 Alnylam reacquires global rights from Sanofi to develop and commercialize patisiran
  • 08 Feb 2018 Patisiran is available under early access or compassionate use in the U.S. and EU
  • 05 Feb 2018 Planned Prescription Drug User Fee Act (PDUFA) date for Amyloidosis in USA (IV) is 2018-08-11
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