Drug Profile


Alternative Names: ARO-AAT

Latest Information Update: 11 Jul 2018

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At a glance

  • Originator Arrowhead Pharmaceuticals
  • Class 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 - Alpha 1-antitrypsin deficiency
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Alpha 1-antitrypsin deficiency

Most Recent Events

  • 29 Jun 2018 Efficacy, adverse events and pharmacodynamics data from a phase I trial in Alpha 1-antitrypsin deficiency released by Arrowhead Pharmaceuticals
  • 27 Jun 2018 The EMA's Committee for Orphan Medicinal Products grants a positive opinion for Orphan drug status to ARO AAT for the treatment of Alpha-1 antitrypsin deficiency
  • 18 Jun 2018 Arrowhead Pharmaceuticals completes enrolment in phase I trial for Alpha 1-antitrypsin deficiency (In Volunteers) in New Zealand (NCT03362242)
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