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Agazisiran - ADARx Pharmaceuticals

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Drug Profile

Agazisiran - ADARx Pharmaceuticals

Alternative Names: ADX-038

Latest Information Update: 28 Aug 2026

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At a glance

  • Originator ADARx Pharmaceuticals
  • Class Eye disorder therapies; Oligonucleotides; Small interfering RNA
  • Mechanism of Action Complement factor B expression inhibitors; RNA interference
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Dry age-related macular degeneration; IgA nephropathy; Membranoproliferative glomerulonephritis
  • Phase I Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 28 Aug 2026 No recent reports of development identified for phase-I development in Paroxysmal-nocturnal-haemoglobinuria(In volunteers) in Australia
  • 14 Apr 2026 Phase-II clinical trials in IgA nephropathy (Combination therapy) in China (Parenteral) (NCT07522099)
  • 14 Apr 2026 Phase-II clinical trials in IgA nephropathy (Monotherapy) in China (Parenteral) (NCT07522099)

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