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ARC AAT

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Drug Profile

ARC AAT

Alternative Names: ARC-AAT

Latest Information Update: 30 Oct 2021

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At a glance

  • Originator Arrowhead Research Corporation
  • Developer 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

  • Discontinued Alpha 1-antitrypsin deficiency

Most Recent Events

  • 02 Dec 2016 Arrowhead Pharmaceuticals withdraws prior to enrolment a phase II trial in Alpha 1-antitrypsin deficiency in Sweden, Italy, Ireland and Canada (IV) (NCT02900183)
  • 29 Nov 2016 Discontinued - Phase-I for Alpha 1-antitrypsin deficiency in Australia, Germany, Netherlands and United Kingdom (IV) due to company shifting its focus on on the development of RNAi therapeutics utilising its subcutaneous (subQ) and extra-hepatic delivery systems
  • 29 Nov 2016 Discontinued - Phase-II for Alpha 1-antitrypsin deficiency in Sweden, Canada, Italy, Ireland (IV) due to company shifting its focus on on the development of RNAi therapeutics utilising its subcutaneous (subQ) and extra-hepatic delivery systems

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